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3. Ligand-dependent hedgehog signaling maintains an undifferentiated, malignant osteosarcoma phenotype

Author

Vaghjiani, Vijesh G., Cochrane, Catherine R., Jayasekara, W. Samantha N., Chong, Wai Chin, Szczepny, Anette, Kumar, Beena, Martelotto, Luciano G., McCaw, Andrew, Carey, Kirstyn, Kansara, Maya, Thomas, David M., Walkley, Carl, Mudge, Stuart, Gough, Daniel J., Downie, Peter A., Peacock, Craig D., Matsui, William, Watkins, D. Neil, and Cain, Jason E.

Published
2023
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5. Generation and multi-dimensional profiling of a childhood cancer cell line atlas defines new therapeutic opportunities

Author

Sun, Claire Xin, Daniel, Paul, Bradshaw, Gabrielle, Shi, Hui, Loi, Melissa, Chew, Nicole, Parackal, Sarah, Tsui, Vanessa, Liang, Yuqing, Koptyra, Mateusz, Adjumain, Shazia, Sun, Christie, Chong, Wai Chin, Fernando, Dasun, Drinkwater, Caroline, Tourchi, Motahhareh, Habarakada, Dilru, Sooraj, Dhanya, Carvalho, Diana, Storm, Phillip B., Baubet, Valerie, Sayles, Leanne C., Fernandez, Elisabet, Nguyen, Thy, Pörksen, Mia, Doan, Anh, Crombie, Duncan E., Panday, Monty, Zhukova, Nataliya, Dun, Matthew D., Ludlow, Louise E., Day, Bryan, Stringer, Brett W., Neeman, Naama, Rubens, Jeffrey A., Raabe, Eric H., Vinci, Maria, Tyrrell, Vanessa, Fletcher, Jamie I., Ekert, Paul G., Dumevska, Biljana, Ziegler, David S., Tsoli, Maria, Syed Sulaiman, Nur Farhana, Loh, Amos Hong Pheng, Low, Sharon Yin Yee, Sweet-Cordero, E. Alejandro, Monje, Michelle, Resnick, Adam, Jones, Chris, Downie, Peter, Williams, Bryan, Rosenbluh, Joseph, Gough, Daniel, Cain, Jason E., and Firestein, Ron

Published
2023
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6. Implementation of DNA Methylation Array Profiling in Pediatric Central Nervous System Tumors: The AIM BRAIN Project: An Australian and New Zealand Children's Hematology and Oncology Group Study

Author

White, Christine L., Kinross, Kathryn M., Buntine, Molly K., Rasouli, Elnaz, Strong, Robyn, Jones, Janelle M., Cain, Jason E., Sturm, Dominik, Sahm, Felix, Jones, David T.W., Pfister, Stefan M., Robertson, Thomas, D'Arcy, Colleen, Rodriguez, Michael L., Dyke, Jason M., Junckerstorff, Reimar, Bhuva, Dharmesh D., Davis, Melissa J., Wood, Paul, Hassall, Tim, Ziegler, David S., Kellie, Stewart, McCowage, Geoffrey, Alvaro, Frank, Kirby, Maria, Heath, John A., Tsui, Karen, Dodgshun, Andrew, Eisenstat, David D., Khuong-Quang, Dong-Anh, Wall, Meaghan, Algar, Elizabeth M., Gottardo, Nicholas G., and Hansford, Jordan R.

Published
2023
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10. PI3K/mTOR is a therapeutically targetable genetic dependency in diffuse intrinsic pontine glioma

Author

Duchatel, Ryan J., primary, Jackson, Evangeline R., additional, Parackal, Sarah G., additional, Kiltschewskij, Dylan, additional, Findlay, Izac J., additional, Mannan, Abdul, additional, Staudt, Dilana E., additional, Thomas, Bryce C., additional, Germon, Zacary P., additional, Laternser, Sandra, additional, Kearney, Padraic S., additional, Jamaluddin, M. Fairuz B., additional, Douglas, Alicia M., additional, Beitaki, Tyrone S., additional, McEwen, Holly P., additional, Persson, Mika L., additional, Hocke, Emily A., additional, Jain, Vaibhav, additional, Aksu, Michael, additional, Manning, Elizabeth E., additional, Murray, Heather C., additional, Verrills, Nicole M., additional, Sun, Claire Xin, additional, Daniel, Paul, additional, Vilain, Ricardo E., additional, Skerrett-Byrne, David A., additional, Nixon, Brett, additional, Hua, Susan, additional, de Bock, Charles E., additional, Colino-Sanguino, Yolanda, additional, Valdes-Mora, Fatima, additional, Tsoli, Maria, additional, Ziegler, David S., additional, Cairns, Murray J., additional, Raabe, Eric H., additional, Vitanza, Nicholas A., additional, Hulleman, Esther, additional, Phoenix, Timothy N., additional, Koschmann, Carl, additional, Alvaro, Frank, additional, Dayas, Christopher V., additional, Tinkle, Christopher L., additional, Wheeler, Helen, additional, Whittle, James R., additional, Eisenstat, David D., additional, Firestein, Ron, additional, Mueller, Sabine, additional, Valvi, Santosh, additional, Hansford, Jordan R., additional, Ashley, David M., additional, Gregory, Simon G., additional, Kilburn, Lindsay B., additional, Nazarian, Javad, additional, Cain, Jason E., additional, and Dun, Matthew D., additional

Published
2024
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11. Trp53 and Rb1 regulate autophagy and ligand-dependent Hedgehog signaling

Author

Cochrane, Catherine R., Vaghjiani, Vijesh, Szczepny, Anette, Jayasekara, W. Samantha N., Gonzalez-Rajal, Alvaro, Kikuchi, Kazu, McCaughan, Geoffrey W., Burgess, Andrew, Gough, Daniel J., Watkins, D. Neil, and Cain, Jason E.

Subjects
Thermo Fisher Scientific Inc., Lung cancer -- Genetic aspects, Tumor proteins -- Genetic aspects, Transcription (Genetics) -- Genetic aspects, Health care industry
Abstract

Ligand-dependent activation of Hedgehog (Hh) signaling in cancer occurs without mutations in canonical pathway genes. Consequently, the genetic basis of Hh pathway activation in adult solid tumors, such as small-cell lung cancer (SCLC), is unknown. Here we show that combined inactivation of Trp53 and Rb1, a defining genetic feature of SCLC, leads to hypersensitivity to Hh ligand in vitro, and during neural tube development in vivo. This response is associated with the aberrant formation of primary cilia, an organelle essential for canonical Hh signaling through smoothened, a transmembrane protein targeted by small-molecule Hh inhibitors. We further show that loss of both Trp53 and Rb1 disables transcription of genes in the autophagic machinery necessary for the degradation of primary cilia. In turn, we also demonstrate a requirement for Kif3a, a gene essential for the formation of primary cilia, in a mouse model of SCLC induced by conditional deletion of both Trp53 and Rb1 in the adult airway. Our results provide a mechanistic framework for therapeutic targeting of ligand-dependent Hh signaling in human cancers with somatic mutations in both TP53 and RB1., Introduction Hedgehog (Hh) signaling is an evolutionarily conserved pathway essential for axial patterning and cell fate determination in development (1). In mammals, Hh signaling is driven by 3 ligands, sonic [...]

Published
2020
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12. Table S4 from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

Published
2023
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13. Supplementary Figures 1-15 from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

Published
2023
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14. Supplementary Data S2 from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

Published
2023
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15. Data from Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, primary, Ji, Sunjong, primary, Waszak, Sebastian M., primary, Sweha, Stefan R., primary, Mota, Mateus, primary, Pun, Matthew, primary, Deogharkar, Akash, primary, Chung, Chan, primary, Tarapore, Rohinton S., primary, Ramage, Samuel, primary, Chi, Andrew, primary, Wen, Patrick Y., primary, Arrillaga-Romany, Isabel, primary, Batchelor, Tracy T., primary, Butowski, Nicholas A., primary, Sumrall, Ashley, primary, Shonka, Nicole, primary, Harrison, Rebecca A., primary, de Groot, John, primary, Mehta, Minesh, primary, Hall, Matthew D., primary, Daghistani, Doured, primary, Cloughesy, Timothy F., primary, Ellingson, Benjamin M., primary, Beccaria, Kevin, primary, Varlet, Pascale, primary, Kim, Michelle M., primary, Umemura, Yoshie, primary, Garton, Hugh, primary, Franson, Andrea, primary, Schwartz, Jonathan, primary, Jain, Rajan, primary, Kachman, Maureen, primary, Baum, Heidi, primary, Burant, Charles F., primary, Mottl, Sophie L., primary, Cartaxo, Rodrigo T., primary, John, Vishal, primary, Messinger, Dana, primary, Qin, Tingting, primary, Peterson, Erik, primary, Sajjakulnukit, Peter, primary, Ravi, Karthik, primary, Waugh, Alyssa, primary, Walling, Dustin, primary, Ding, Yujie, primary, Xia, Ziyun, primary, Schwendeman, Anna, primary, Hawes, Debra, primary, Yang, Fusheng, primary, Judkins, Alexander R., primary, Wahl, Daniel, primary, Lyssiotis, Costas A., primary, de la Nava, Daniel, primary, Alonso, Marta M., primary, Eze, Augustine, primary, Spitzer, Jasper, primary, Schmidt, Susanne V., primary, Duchatel, Ryan J., primary, Dun, Matthew D., primary, Cain, Jason E., primary, Jiang, Li, primary, Stopka, Sylwia A., primary, Baquer, Gerard, primary, Regan, Michael S., primary, Filbin, Mariella G., primary, Agar, Nathalie Y.R., primary, Zhao, Lili, primary, Kumar-Sinha, Chandan, primary, Mody, Rajen, primary, Chinnaiyan, Arul, primary, Kurokawa, Ryo, primary, Pratt, Drew, primary, Yadav, Viveka N., primary, Grill, Jacques, primary, Kline, Cassie, primary, Mueller, Sabine, primary, Resnick, Adam, primary, Nazarian, Javad, primary, Allen, Joshua E., primary, Odia, Yazmin, primary, Gardner, Sharon L., primary, and Koschmann, Carl, primary

Published
2023
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16. Deep multi-region whole-genome sequencing reveals heterogeneity and gene-by-environment interactions in treatment-naive, metastatic lung cancer

Author

Leong, Tracy L., Gayevskiy, Velimir, Steinfort, Daniel P., De Massy, Marc R., Gonzalez-Rajal, Alvaro, Marini, Kieren D., Stone, Emily, Chin, Venessa, Havryk, Adrian, Plit, Marshall, Irving, Louis B., Jennings, Barton R., McCloy, Rachael A., Jayasekara, W. Samantha N., Alamgeer, Muhammad, Boolell, Vishal, Field, Andrew, Russell, Prudence A., Kumar, Beena, Gough, Daniel J., Szczepny, Anette, Ganju, Vinod, Rossello, Fernando J., Cain, Jason E., Papenfuss, Anthony T., Asselin-Labat, Marie-Liesse, Cowley, Mark J., and Watkins, D. Neil

Published
2019
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17. Clinical Efficacy of ONC201 in H3K27M-Mutant Diffuse Midline Gliomas Is Driven by Disruption of Integrated Metabolic and Epigenetic Pathways

Author

Venneti, Sriram, primary, Kawakibi, Abed Rahman, additional, Ji, Sunjong, additional, Waszak, Sebastian M., additional, Sweha, Stefan R., additional, Mota, Mateus, additional, Pun, Matthew, additional, Deogharkar, Akash, additional, Chung, Chan, additional, Tarapore, Rohinton S., additional, Ramage, Samuel, additional, Chi, Andrew, additional, Wen, Patrick Y., additional, Arrillaga-Romany, Isabel, additional, Batchelor, Tracy T., additional, Butowski, Nicholas A., additional, Sumrall, Ashley, additional, Shonka, Nicole, additional, Harrison, Rebecca A., additional, de Groot, John, additional, Mehta, Minesh, additional, Hall, Matthew D., additional, Daghistani, Doured, additional, Cloughesy, Timothy F., additional, Ellingson, Benjamin M., additional, Beccaria, Kevin, additional, Varlet, Pascale, additional, Kim, Michelle M., additional, Umemura, Yoshie, additional, Garton, Hugh, additional, Franson, Andrea, additional, Schwartz, Jonathan, additional, Jain, Rajan, additional, Kachman, Maureen, additional, Baum, Heidi, additional, Burant, Charles F., additional, Mottl, Sophie L., additional, Cartaxo, Rodrigo T., additional, John, Vishal, additional, Messinger, Dana, additional, Qin, Tingting, additional, Peterson, Erik, additional, Sajjakulnukit, Peter, additional, Ravi, Karthik, additional, Waugh, Alyssa, additional, Walling, Dustin, additional, Ding, Yujie, additional, Xia, Ziyun, additional, Schwendeman, Anna, additional, Hawes, Debra, additional, Yang, Fusheng, additional, Judkins, Alexander R., additional, Wahl, Daniel, additional, Lyssiotis, Costas A., additional, de la Nava, Daniel, additional, Alonso, Marta M., additional, Eze, Augustine, additional, Spitzer, Jasper, additional, Schmidt, Susanne V., additional, Duchatel, Ryan J., additional, Dun, Matthew D., additional, Cain, Jason E., additional, Jiang, Li, additional, Stopka, Sylwia A., additional, Baquer, Gerard, additional, Regan, Michael S., additional, Filbin, Mariella G., additional, Agar, Nathalie Y.R., additional, Zhao, Lili, additional, Kumar-Sinha, Chandan, additional, Mody, Rajen, additional, Chinnaiyan, Arul, additional, Kurokawa, Ryo, additional, Pratt, Drew, additional, Yadav, Viveka N., additional, Grill, Jacques, additional, Kline, Cassie, additional, Mueller, Sabine, additional, Resnick, Adam, additional, Nazarian, Javad, additional, Allen, Joshua E., additional, Odia, Yazmin, additional, Gardner, Sharon L., additional, and Koschmann, Carl, additional

Published
2023
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18. SYST-15 GENERATION AND MULTI-DIMENSIONAL PROFILING OF A CHILDHOOD CANCER CELL LINE ATLAS DEFINES NEW THERAPEUTIC OPPORTUNITIES

Author

Daniel, Paul, primary, Sun, Claire Xin, additional, Bradshaw, Gabrielle, additional, Shi, Hui, additional, Loi, Melissa, additional, Chew, Nicole, additional, Parackal, Sarah, additional, Tsui, Vanessa, additional, Liang, Yuqing, additional, Koptyra, Mateusz, additional, Adjumain, Shazia, additional, Sun, Christie, additional, Chong, Wai Chin, additional, Fernando, Dasun, additional, Drinkwater, Caroline, additional, Tourchi, Motahhareh, additional, Habarakada, Dilru, additional, Carvalho, Diana, additional, Storm, Phillip B, additional, Baubet, Valerie, additional, Sayles, Leanne C, additional, Fernandez, Elisabet, additional, Zhukova, Nataliya, additional, Dun, Matthew D, additional, Ludlow, Louise E, additional, Day, Bryan, additional, Stringer, Brett W, additional, Neeman, Naama, additional, Rubens, Jeffrey A, additional, Raabe, Eric H, additional, Vinci, Maria, additional, Tyrrell, Vanessa, additional, Fletcher, Jamie I, additional, Ekert, Paul G, additional, Dumevska, Biljana, additional, Ziegler, David S, additional, Tsoli, Maria, additional, Sulaiman, Nur Farhana Syed, additional, Loh, Amos Hong Pheng, additional, Low, Sharon Yin Yee, additional, Sweet-Cordero, E Alejandro, additional, Monje, Michelle, additional, Resnick, Adam, additional, Jones, Chris, additional, Downie, Peter, additional, Williams, Bryan, additional, Rosenbluh, Joseph, additional, Gough, Daniel, additional, Cain, Jason E, additional, and Firestein, Ron, additional

Published
2023
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19. Supplementary Data from ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

Author

Jackson, Evangeline R., primary, Duchatel, Ryan J., primary, Staudt, Dilana E., primary, Persson, Mika L., primary, Mannan, Abdul, primary, Yadavilli, Sridevi, primary, Parackal, Sarah, primary, Game, Shaye, primary, Chong, Wai Chin, primary, Jayasekara, W. Samantha N., primary, Grand, Marion Le, primary, Kearney, Padraic S., primary, Douglas, Alicia M., primary, Findlay, Izac J., primary, Germon, Zacary P., primary, McEwen, Holly P., primary, Beitaki, Tyrone S., primary, Patabendige, Adjanie, primary, Skerrett-Byrne, David A., primary, Nixon, Brett, primary, Smith, Nathan D., primary, Day, Bryan, primary, Manoharan, Neevika, primary, Nagabushan, Sumanth, primary, Hansford, Jordan R., primary, Govender, Dinisha, primary, McCowage, Geoff B., primary, Firestein, Ron, primary, Howlett, Meegan, primary, Endersby, Raelene, primary, Gottardo, Nicholas G., primary, Alvaro, Frank, primary, Waszak, Sebastian M., primary, Larsen, Martin R., primary, Colino-Sanguino, Yolanda, primary, Valdes-Mora, Fatima, primary, Rakotomalala, Andria, primary, Meignan, Samuel, primary, Pasquier, Eddy, primary, André, Nicolas, primary, Hulleman, Esther, primary, Eisenstat, David D., primary, Vitanza, Nicholas A., primary, Nazarian, Javad, primary, Koschmann, Carl, primary, Mueller, Sabine, primary, Cain, Jason E., primary, and Dun, Matthew D., primary

Published
2023
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20. Data from ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

Author

Jackson, Evangeline R., primary, Duchatel, Ryan J., primary, Staudt, Dilana E., primary, Persson, Mika L., primary, Mannan, Abdul, primary, Yadavilli, Sridevi, primary, Parackal, Sarah, primary, Game, Shaye, primary, Chong, Wai Chin, primary, Jayasekara, W. Samantha N., primary, Grand, Marion Le, primary, Kearney, Padraic S., primary, Douglas, Alicia M., primary, Findlay, Izac J., primary, Germon, Zacary P., primary, McEwen, Holly P., primary, Beitaki, Tyrone S., primary, Patabendige, Adjanie, primary, Skerrett-Byrne, David A., primary, Nixon, Brett, primary, Smith, Nathan D., primary, Day, Bryan, primary, Manoharan, Neevika, primary, Nagabushan, Sumanth, primary, Hansford, Jordan R., primary, Govender, Dinisha, primary, McCowage, Geoff B., primary, Firestein, Ron, primary, Howlett, Meegan, primary, Endersby, Raelene, primary, Gottardo, Nicholas G., primary, Alvaro, Frank, primary, Waszak, Sebastian M., primary, Larsen, Martin R., primary, Colino-Sanguino, Yolanda, primary, Valdes-Mora, Fatima, primary, Rakotomalala, Andria, primary, Meignan, Samuel, primary, Pasquier, Eddy, primary, André, Nicolas, primary, Hulleman, Esther, primary, Eisenstat, David D., primary, Vitanza, Nicholas A., primary, Nazarian, Javad, primary, Koschmann, Carl, primary, Mueller, Sabine, primary, Cain, Jason E., primary, and Dun, Matthew D., primary

Published
2023
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21. The tumor suppressor Hic1 maintains chromosomal stability independent of Tp53

Author

Szczepny, Anette, Carey, Kirstyn, McKenzie, Lisa, Jayasekara, W. Samantha N., Rossello, Fernando, Gonzalez-Rajal, Alvaro, McCaw, Andrew S., Popovski, Dean, Wang, Die, Sadler, Anthony J., Mahar, Annabelle, Russell, Prudence A., Wright, Gavin, McCloy, Rachael A., Garama, Daniel J., Gough, Daniel J., Baylin, Stephen B., Burgess, Andrew, Cain, Jason E., and Watkins, D. Neil

Published
2018
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22. ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma

Author

Jackson, Evangeline R., primary, Duchatel, Ryan J., additional, Staudt, Dilana E., additional, Persson, Mika L., additional, Mannan, Abdul, additional, Yadavilli, Sridevi, additional, Parackal, Sarah, additional, Game, Shaye, additional, Chong, Wai Chin, additional, Jayasekara, W. Samantha N., additional, Grand, Marion Le, additional, Kearney, Padraic S., additional, Douglas, Alicia M., additional, Findlay, Izac J., additional, Germon, Zacary P., additional, McEwen, Holly P., additional, Beitaki, Tyrone S., additional, Patabendige, Adjanie, additional, Skerrett-Byrne, David A., additional, Nixon, Brett, additional, Smith, Nathan D., additional, Day, Bryan, additional, Manoharan, Neevika, additional, Nagabushan, Sumanth, additional, Hansford, Jordan R., additional, Govender, Dinisha, additional, McCowage, Geoff B., additional, Firestein, Ron, additional, Howlett, Meegan, additional, Endersby, Raelene, additional, Gottardo, Nicholas G., additional, Alvaro, Frank, additional, Waszak, Sebastian M., additional, Larsen, Martin R., additional, Colino-Sanguino, Yolanda, additional, Valdes-Mora, Fatima, additional, Rakotomalala, Andria, additional, Meignan, Samuel, additional, Pasquier, Eddy, additional, André, Nicolas, additional, Hulleman, Esther, additional, Eisenstat, David D., additional, Vitanza, Nicholas A., additional, Nazarian, Javad, additional, Koschmann, Carl, additional, Mueller, Sabine, additional, Cain, Jason E., additional, and Dun, Matthew D., additional

Published
2023
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24. Data from Phosphoproteomic Profiling Reveals ALK and MET as Novel Actionable Targets across Synovial Sarcoma Subtypes

Author

Fleuren, Emmy D.G., primary, Vlenterie, Myrella, primary, van der Graaf, Winette T.A., primary, Hillebrandt-Roeffen, Melissa H.S., primary, Blackburn, James, primary, Ma, Xiuquan, primary, Chan, Howard, primary, Magias, Mandy C., primary, van Erp, Anke, primary, van Houdt, Laurens, primary, Cebeci, Sabri A.S., primary, van de Ven, Amy, primary, Flucke, Uta E., primary, Heyer, Erin E., primary, Thomas, David M., primary, Lord, Christopher J., primary, Marini, Kieren D., primary, Vaghjiani, Vijesh, primary, Mercer, Tim R., primary, Cain, Jason E., primary, Wu, Jianmin, primary, Versleijen-Jonkers, Yvonne M.H., primary, and Daly, Roger J., primary

Published
2023
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25. Supplemental Table 4 from Phosphoproteomic Profiling Reveals ALK and MET as Novel Actionable Targets across Synovial Sarcoma Subtypes

Author

Fleuren, Emmy D.G., primary, Vlenterie, Myrella, primary, van der Graaf, Winette T.A., primary, Hillebrandt-Roeffen, Melissa H.S., primary, Blackburn, James, primary, Ma, Xiuquan, primary, Chan, Howard, primary, Magias, Mandy C., primary, van Erp, Anke, primary, van Houdt, Laurens, primary, Cebeci, Sabri A.S., primary, van de Ven, Amy, primary, Flucke, Uta E., primary, Heyer, Erin E., primary, Thomas, David M., primary, Lord, Christopher J., primary, Marini, Kieren D., primary, Vaghjiani, Vijesh, primary, Mercer, Tim R., primary, Cain, Jason E., primary, Wu, Jianmin, primary, Versleijen-Jonkers, Yvonne M.H., primary, and Daly, Roger J., primary

Published
2023
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26. Supplementary Tables 1-3 and Figures 1-9 from Phosphoproteomic Profiling Reveals ALK and MET as Novel Actionable Targets across Synovial Sarcoma Subtypes

Author

Fleuren, Emmy D.G., primary, Vlenterie, Myrella, primary, van der Graaf, Winette T.A., primary, Hillebrandt-Roeffen, Melissa H.S., primary, Blackburn, James, primary, Ma, Xiuquan, primary, Chan, Howard, primary, Magias, Mandy C., primary, van Erp, Anke, primary, van Houdt, Laurens, primary, Cebeci, Sabri A.S., primary, van de Ven, Amy, primary, Flucke, Uta E., primary, Heyer, Erin E., primary, Thomas, David M., primary, Lord, Christopher J., primary, Marini, Kieren D., primary, Vaghjiani, Vijesh, primary, Mercer, Tim R., primary, Cain, Jason E., primary, Wu, Jianmin, primary, Versleijen-Jonkers, Yvonne M.H., primary, and Daly, Roger J., primary

Published
2023
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27. Supplemental Table 5 from Phosphoproteomic Profiling Reveals ALK and MET as Novel Actionable Targets across Synovial Sarcoma Subtypes

Author

Fleuren, Emmy D.G., primary, Vlenterie, Myrella, primary, van der Graaf, Winette T.A., primary, Hillebrandt-Roeffen, Melissa H.S., primary, Blackburn, James, primary, Ma, Xiuquan, primary, Chan, Howard, primary, Magias, Mandy C., primary, van Erp, Anke, primary, van Houdt, Laurens, primary, Cebeci, Sabri A.S., primary, van de Ven, Amy, primary, Flucke, Uta E., primary, Heyer, Erin E., primary, Thomas, David M., primary, Lord, Christopher J., primary, Marini, Kieren D., primary, Vaghjiani, Vijesh, primary, Mercer, Tim R., primary, Cain, Jason E., primary, Wu, Jianmin, primary, Versleijen-Jonkers, Yvonne M.H., primary, and Daly, Roger J., primary

Published
2023
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29. ADAM17 selectively activates the IL‐6 trans‐signaling/ERK MAPK axis in KRAS‐addicted lung cancer

Author

Saad, Mohamed I, Alhayyani, Sultan, McLeod, Louise, Yu, Liang, Alanazi, Mohammad, Deswaerte, Virginie, Tang, Ke, Jarde, Thierry, Smith, Julian A, Prodanovic, Zdenka, Tate, Michelle D, Balic, Jesse J, Watkins, D Neil, Cain, Jason E, Bozinovski, Steven, Algar, Elizabeth, Kohmoto, Tomohiro, Ebi, Hiromichi, Ferlin, Walter, Garbers, Christoph, Ruwanpura, Saleela, Sagi, Irit, Rose‐John, Stefan, and Jenkins, Brendan J

Published
2019
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32. Imipridones affect tumor bioenergetics and promote cell lineage differentiation in diffuse midline gliomas

Author

Przystal, Justyna M, Cianciolo Cosentino, Chiara, Yadavilli, Sridevi, Zhang, Jie, Laternser, Sandra, Bonner, Erin R, Prasad, Rachna, Dawood, Adam A, Lobeto, Nina, Chin Chong, Wai, Biery, Matt C, Myers, Carrie, Olson, James M, Panditharatna, Eshini, Kritzer, Bettina, Mourabit, Sulayman, Vitanza, Nicholas A, Filbin, Mariella G, de Iuliis, Geoffry N, Dun, Matthew D, Koschmann, Carl, Cain, Jason E, Grotzer, Michael A, Waszak, Sebastian M, Mueller, Sabine, Nazarian, Javad, and University of Zurich

Subjects
Cancer Research, Pyridines, Imidazoles, 610 Medicine & health, Antineoplastic Agents, Glioma, Heterocyclic Compounds, 4 or More Rings, Mice, 2728 Neurology (clinical), Pyrimidines, Oncology, 10036 Medical Clinic, Cell Line, Tumor, Basic and Translational Investigations, Animals, Humans, 2730 Oncology, 1306 Cancer Research, Cell Lineage, Neurology (clinical), Child, Energy Metabolism, Zebrafish
Abstract

Background Pediatric diffuse midline gliomas (DMGs) are incurable childhood cancers. The imipridone ONC201 has shown early clinical efficacy in a subset of DMGs. However, the anticancer mechanisms of ONC201 and its derivative ONC206 have not been fully described in DMGs. Methods DMG models including primary human in vitro (n = 18) and in vivo (murine and zebrafish) models, and patient (n = 20) frozen and FFPE specimens were used. Drug-target engagement was evaluated using in silico ChemPLP and in vitro thermal shift assay. Drug toxicity and neurotoxicity were assessed in zebrafish models. Seahorse XF Cell Mito Stress Test, MitoSOX and TMRM assays, and electron microscopy imaging were used to assess metabolic signatures. Cell lineage differentiation and drug-altered pathways were defined using bulk and single-cell RNA-seq. Results ONC201 and ONC206 reduce viability of DMG cells in nM concentrations and extend survival of DMG PDX models (ONC201: 117 days, P = .01; ONC206: 113 days, P = .001). ONC206 is 10X more potent than ONC201 in vitro and combination treatment was the most efficacious at prolonging survival in vivo (125 days, P = .02). Thermal shift assay confirmed that both drugs bind to ClpP, with ONC206 exhibiting a higher binding affinity as assessed by in silico ChemPLP. ClpP activation by both drugs results in impaired tumor cell metabolism, mitochondrial damage, ROS production, activation of integrative stress response (ISR), and apoptosis in vitro and in vivo. Strikingly, imipridone treatment triggered a lineage shift from a proliferative, oligodendrocyte precursor-like state to a mature, astrocyte-like state. Conclusion Targeting mitochondrial metabolism and ISR activation effectively impairs DMG tumorigenicity. These results supported the initiation of two pediatric clinical trials (NCT05009992, NCT04732065).

Published
2022

33. Imipridones affect tumor bioenergetics and promote cell lineage differentiation in diffuse midline gliomas

Author

Przystal, Justyna M, primary, Cianciolo Cosentino, Chiara, additional, Yadavilli, Sridevi, additional, Zhang, Jie, additional, Laternser, Sandra, additional, Bonner, Erin R, additional, Prasad, Rachna, additional, Dawood, Adam A, additional, Lobeto, Nina, additional, Chin Chong, Wai, additional, Biery, Matt C, additional, Myers, Carrie, additional, Olson, James M, additional, Panditharatna, Eshini, additional, Kritzer, Bettina, additional, Mourabit, Sulayman, additional, Vitanza, Nicholas A, additional, Filbin, Mariella G, additional, de Iuliis, Geoffry N, additional, Dun, Matthew D, additional, Koschmann, Carl, additional, Cain, Jason E, additional, Grotzer, Michael A, additional, Waszak, Sebastian M, additional, Mueller, Sabine, additional, and Nazarian, Javad, additional

Published
2022
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36. Clinical phenotypes and prognostic features of embryonal tumours with multi-layered rosettes: a Rare Brain Tumor Registry study

Author

Khan, Sara, primary, Solano-Paez, Palma, additional, Suwal, Tannu, additional, Lu, Mei, additional, Al-Karmi, Salma, additional, Ho, Ben, additional, Mumal, Iqra, additional, Shago, Mary, additional, Hoffman, Lindsey M, additional, Dodgshun, Andrew, additional, Nobusawa, Sumihito, additional, Tabori, Uri, additional, Bartels, Ute, additional, Ziegler, David S, additional, Hansford, Jordan R, additional, Ramaswamy, Vijay, additional, Hawkins, Cynthia, additional, Dufour, Christelle, additional, André, Nicolas, additional, Bouffet, Eric, additional, Huang, Annie, additional, Gonzalez CV, Almeida, additional, Stephens, Derek, additional, Leary, Sarah, additional, Marrano, Paula, additional, Fonseca, Adriana, additional, Thacker, Nirav, additional, Li, Bryan K., additional, Lindsay, Holly B., additional, Lassaletta, Alvaro, additional, Bendel, Anne E., additional, Moertel, Christopher, additional, Morales La Madrid, Andres, additional, Santa-Maria, Vicente, additional, Lavarino, Cinzia, additional, Rivas, Eloy, additional, Perreault, Sebastian, additional, Ellezam, Benjamin, additional, Weil, Alexander G, additional, Jabado, Nada, additional, Oviedo, Angelica, additional, Yalon-Oren, Michal, additional, Amariglio, Laura, additional, Toledano, Helen, additional, Dvir, Rina, additional, Loukides, James, additional, Van Meter, Timothy E., additional, Nakamura, Hideo, additional, Wong, Tai-Tong, additional, Wu, Kuo-Sheng, additional, Cheng, Chien-Jui, additional, Ra, Young-Shin, additional, La Spina, Milena, additional, Massimi, Luca, additional, Buccoliero, Anna Maria, additional, Reddy, Alyssa, additional, Li, Rong, additional, Gillespie, G. Yancey, additional, Adamek, Dariusz, additional, Fangusaro, Jason, additional, Scharnhorst, David, additional, Torkildson, Joseph, additional, Johnston, Donna, additional, Michaud, Jean, additional, LafayCousin, Lucie, additional, Chan, Jennifer, additional, Van Landeghem, Frank, additional, Wilson, Beverly, additional, Camelo-Piragua, Sandra, additional, Kabbara, Nabil, additional, Boutarbouch, Mahjouba, additional, Hanson, Derek, additional, Jacobsen, Chad, additional, Wright, Karen, additional, Vibhakar, Rajeev, additional, Levy, Jean M., additional, Wang, Yin, additional, Catchpoole, Daniel, additional, Gerber, Nicholas, additional, Grotzer, Michael A., additional, Shen, Violet, additional, Plant, Ashley, additional, Dunham, Christopher, additional, Joao Gil da Costa, Maria, additional, Ramanujachar, Ramya, additional, Raabe, Eric, additional, Rubens, Jeffery, additional, Phillips, Joanna, additional, Gupta, Nalin, additional, Demir, Haci Ahmet, additional, Dahl, Christine, additional, Jorgensen, Mette, additional, Hwang, Eugene I., additional, Packer, Roger J, additional, Smith, Amy, additional, Tan, Enrica, additional, Low, Sharon, additional, Lu, Jian-Qiang, additional, Ng, Ho- Keung, additional, Kresak, Jesse L., additional, Gururangan, Sridharan, additional, Pomeroy, Scott L., additional, Sirachainan, Nongnuch, additional, Hongeng, Suradej, additional, Magimairajan, Vanan, additional, Sinha, Roona, additional, Mushtaq, Naureen, additional, Antony, Reuben, additional, Sato, Mariko, additional, Samuel, David, additional, Zapotocky, Michal, additional, Afzal, Samina, additional, Walter, Andrew, additional, Tihan, Tarik, additional, Tsang, Derek S., additional, Gajjar, Amar, additional, Wood, Paul, additional, Cain, Jason E., additional, Downie, Peter A., additional, Gottardo, Nicolas, additional, Branson, Helen, additional, Laughlin, Suzanne, additional, Ertl-Wagner, Birgit, additional, Kulkarni, Abhaya V., additional, Taylor, Michael D., additional, Drake, James, additional, Ibrahim, George M., additional, Dirks, Peter B., additional, Rutka, James T., additional, Somers, Gino R., additional, Hazrati, Lili-Naz, additional, Bourdeaut, Franck, additional, Padovani, Laetitia, additional, Grundy, Richard G., additional, Mazewski, Claire M., additional, and Fouladi, Maryam, additional

Published
2021
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37. Lineage-restricted neoplasia driven by Myc defaults to small cell lung cancer when combined with loss of p53 and Rb in the airway epithelium

Author

Chen, Jasmine, primary, Guanizo, Aleks, additional, Luong, Quinton, additional, Jayasekara, W. Samantha N., additional, Jayasinghe, Dhilshan, additional, Inampudi, Chaitanya, additional, Szczepny, Anette, additional, Garama, Daniel J., additional, Russell, Prudence A., additional, Ganju, Vinod, additional, Cain, Jason E., additional, Watkins, D. Neil, additional, and Gough, Daniel J., additional

Published
2021
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38. Atypical Teratoid Rhabdoid Tumours Are Susceptible to Panobinostat-Mediated Differentiation Therapy

Author

Chong, Wai C., primary, Jayasekara, W. Samantha N., additional, Vaghjiani, Vijesh G., additional, Parackal, Sarah, additional, Sun, Claire, additional, Popovski, Dean, additional, Algar, Elizabeth M., additional, Firestein, Ron, additional, Wood, Paul J., additional, Khan, Sara, additional, Huang, Annie, additional, Ashley, David M., additional, Downie, Peter, additional, and Cain, Jason E., additional

Published
2021
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40. Preclinical and clinical evaluation of German-sourced ONC201 for the treatment of H3K27M-mutant diffuse intrinsic pontine glioma

Author

Duchatel, Ryan J, Mannan, Abdul, Woldu, Ameha S, Hawtrey, Tom, Hindley, Phoebe A, Douglas, Alicia M, Jackson, Evangeline R, Findlay, Izac J, Germon, Zacary P, Staudt, Dilana, Kearney, Padraic S, Smith, Nathan D, Hindley, Kate E, Cain, Jason E, André, Nicolas, La Madrid, Andres Morales, Nixon, Brett; https://orcid.org/0000-0003-2745-8188, De Iuliis, Geoffry N, Nazarian, Javad; https://orcid.org/0000-0002-1951-9828, Irish, Kathleen, Alvaro, Frank, Eisenstat, David D, Beck, Alexander, Vitanza, Nicholas A, Mueller, Sabine; https://orcid.org/0000-0002-3452-5150, Morris, Jonathan C, Dun, Matthew D; https://orcid.org/0000-0002-9063-5370, Duchatel, Ryan J, Mannan, Abdul, Woldu, Ameha S, Hawtrey, Tom, Hindley, Phoebe A, Douglas, Alicia M, Jackson, Evangeline R, Findlay, Izac J, Germon, Zacary P, Staudt, Dilana, Kearney, Padraic S, Smith, Nathan D, Hindley, Kate E, Cain, Jason E, André, Nicolas, La Madrid, Andres Morales, Nixon, Brett; https://orcid.org/0000-0003-2745-8188, De Iuliis, Geoffry N, Nazarian, Javad; https://orcid.org/0000-0002-1951-9828, Irish, Kathleen, Alvaro, Frank, Eisenstat, David D, Beck, Alexander, Vitanza, Nicholas A, Mueller, Sabine; https://orcid.org/0000-0002-3452-5150, Morris, Jonathan C, and Dun, Matthew D; https://orcid.org/0000-0002-9063-5370

Abstract

Background Diffuse intrinsic pontine glioma (DIPG) is a fatal childhood brainstem tumor for which radiation is the only treatment. Case studies report a clinical response to ONC201 for patients with H3K27M-mutant gliomas. Oncoceutics (ONC201) is only available in the United States and Japan; however, in Germany, DIPG patients can be prescribed and dispensed a locally produced compound-ONC201 German-sourced ONC201 (GsONC201). Pediatric oncologists face the dilemma of supporting the administration of GsONC201 as conjecture surrounds its authenticity. Therefore, we compared GsONC201 to original ONC201 manufactured by Oncoceutics Inc. Methods Authenticity of GsONC201 was determined by high-resolution mass spectrometry and nuclear magnetic resonance spectroscopy. Biological activity was shown via assessment of on-target effects, in vitro growth, proliferation, and apoptosis analysis. Patient-derived xenograft mouse models were used to assess plasma and brain tissue pharmacokinetics, pharmacodynamics, and overall survival (OS). The clinical experience of 28 H3K27M+ mutant DIPG patients who received GsONC201 (2017-2020) was analyzed. Results GsONC201 harbored the authentic structure, however, was formulated as a free base rather than the dihydrochloride salt used in clinical trials. GsONC201 in vitro and in vivo efficacy and drug bioavailability studies showed no difference compared to Oncoceutics ONC201. Patients treated with GsONC201 (n = 28) showed a median OS of 18 months (P = .0007). GsONC201 patients who underwent reirradiation showed a median OS of 22 months compared to 12 months for GsONC201 patients who did not (P = .012). Conclusions This study confirms the biological activity of GsONC201 and documents the OS of patients who received the drug; however, GsONC201 was never used as a monotherapy.

Published
2021

42. GLI3 repressor controls functional development of the mouse ureter

Author

Cain, Jason E., Islam, Epshita, Haxho, Fiona, Blake, Joshua, and Rosenblum, Norman D.

Subjects
Gene mutations -- Health aspects -- Research -- Genetic aspects -- Physiological aspects, Cell differentiation -- Research -- Genetic aspects -- Health aspects -- Physiological aspects, Health care industry
Abstract

Obstructive and nonobstructive forms of hydronephrosis (increased diameter of the renal pelvis and calyces) and hydroureter (dilatation of the ureter) are the most frequently detected antenatal abnormalities, yet the underlying molecular mechanisms are largely undefined. Hedgehog (Hh) proteins control tissue patterning and cell differentiation by promoting GLI-dependent transcriptional activation and by inhibiting the processing of GLI3 to a transcriptional repressor. Genetic mutations that generate a truncated GLI3 protein similar in size to the repressor in humans with Pallister-Hall syndrome (PHS; a disorder whose characteristics include renal abnormalities) and hydroureter implicate Hh-dependent signaling in ureter morphogenesis and function. Here, we determined that Hh signaling controls 2 cell populations required for the initiation and transmission of coordinated ureter contractions. Tissue-specific inactivation of the Hh cell surface effector Smoothened (Smo) in the renal pelvic and upper ureteric mesenchyme resulted in nonobstructive hydronephrosis and hydroureter characterized by ureter dyskinesia. Mutant mice had reduced expression of markers of cell populations implicated in the coordination of unidirectional ureter peristalsis (specifically, Kit and hyperpolarization-activation cation-3 channel [Hcn3]), but exhibited normal epithelial and smooth muscle cell differentiation. Kit deficiency in a mouse model of PHS suggested a pathogenic role for GLI3 repressor in Smo-deficient embryos; indeed, genetic inactivation of Gli3 in Smo-deficient mice rescued their hydronephrosis, hydroureter, Kit and Hcn3 expression, and ureter peristalsis. Together, these data demonstrate that Hh signaling controls Kit and Hcn3 expression and ureter peristalsis., Introduction Congenital dilatation of the urinary tract, defined as increased diameter of the renal pelvis (hydronephrosis) with or without dilatation of the ureter (hydroureter), is the most frequently detected abnormality [...]

Published
2011
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43. A non-genetic, cell cycle-dependent mechanism of platinum resistance in lung adenocarcinoma

Author

Gonzalez Rajal, Alvaro, primary, Marzec, Kamila A, additional, McCloy, Rachael A, additional, Nobis, Max, additional, Chin, Venessa, additional, Hastings, Jordan F, additional, Lai, Kaitao, additional, Kennerson, Marina, additional, Hughes, William E, additional, Vaghjiani, Vijesh, additional, Timpson, Paul, additional, Cain, Jason E, additional, Watkins, D Neil, additional, Croucher, David R, additional, and Burgess, Andrew, additional

Published
2021
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44. Author response: A non-genetic, cell cycle-dependent mechanism of platinum resistance in lung adenocarcinoma

Author

Gonzalez Rajal, Alvaro, primary, Marzec, Kamila A, additional, McCloy, Rachael A, additional, Nobis, Max, additional, Chin, Venessa, additional, Hastings, Jordan F, additional, Lai, Kaitao, additional, Kennerson, Marina, additional, Hughes, William E, additional, Vaghjiani, Vijesh, additional, Timpson, Paul, additional, Cain, Jason E, additional, Watkins, D Neil, additional, Croucher, David R, additional, and Burgess, Andrew, additional

Published
2021
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45. Preclinical and clinical evaluation of German-sourced ONC201 for the treatment of H3K27M-mutant diffuse intrinsic pontine glioma

Author

Duchatel, Ryan J, primary, Mannan, Abdul, additional, Woldu, Ameha S, additional, Hawtrey, Tom, additional, Hindley, Phoebe A, additional, Douglas, Alicia M, additional, Jackson, Evangeline R, additional, Findlay, Izac J, additional, Germon, Zacary P, additional, Staudt, Dilana, additional, Kearney, Padraic S, additional, Smith, Nathan D, additional, Hindley, Kate E, additional, Cain, Jason E, additional, André, Nicolas, additional, La Madrid, Andres Morales, additional, Nixon, Brett, additional, De Iuliis, Geoffry N, additional, Nazarian, Javad, additional, Irish, Kathleen, additional, Alvaro, Frank, additional, Eisenstat, David D, additional, Beck, Alexander, additional, Vitanza, Nicholas A, additional, Mueller, Sabine, additional, Morris, Jonathan C, additional, and Dun, Matthew D, additional

Published
2021
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47. Integrated Proteogenomic Characterization across Major Histological Types of Pediatric Brain Cancer

Author

Petralia, Francesca, primary, Tignor, Nicole, additional, Reva, Boris, additional, Koptyra, Mateusz, additional, Chowdhury, Shrabanti, additional, Rykunov, Dmitry, additional, Krek, Azra, additional, Ma, Weiping, additional, Zhu, Yuankun, additional, Ji, Jiayi, additional, Calinawan, Anna, additional, Whiteaker, Jeffrey R., additional, Colaprico, Antonio, additional, Stathias, Vasileios, additional, Omelchenko, Tatiana, additional, Song, Xiaoyu, additional, Raman, Pichai, additional, Guo, Yiran, additional, Brown, Miguel A., additional, Ivey, Richard G., additional, Szpyt, John, additional, Guha Thakurta, Sanjukta, additional, Gritsenko, Marina A., additional, Weitz, Karl K., additional, Lopez, Gonzalo, additional, Kalayci, Selim, additional, Gümüş, Zeynep H., additional, Yoo, Seungyeul, additional, da Veiga Leprevost, Felipe, additional, Chang, Hui-Yin, additional, Krug, Karsten, additional, Katsnelson, Lizabeth, additional, Wang, Ying, additional, Kennedy, Jacob J., additional, Voytovich, Uliana J., additional, Zhao, Lei, additional, Gaonkar, Krutika S., additional, Ennis, Brian M., additional, Zhang, Bo, additional, Baubet, Valerie, additional, Tauhid, Lamiya, additional, Lilly, Jena V., additional, Mason, Jennifer L., additional, Farrow, Bailey, additional, Young, Nathan, additional, Leary, Sarah, additional, Moon, Jamie, additional, Petyuk, Vladislav A., additional, Nazarian, Javad, additional, Adappa, Nithin D., additional, Palmer, James N., additional, Lober, Robert M., additional, Rivero-Hinojosa, Samuel, additional, Wang, Liang-Bo, additional, Wang, Joshua M., additional, Broberg, Matilda, additional, Chu, Rosalie K., additional, Moore, Ronald J., additional, Monroe, Matthew E., additional, Zhao, Rui, additional, Smith, Richard D., additional, Zhu, Jun, additional, Robles, Ana I., additional, Mesri, Mehdi, additional, Boja, Emily, additional, Hiltke, Tara, additional, Rodriguez, Henry, additional, Zhang, Bing, additional, Schadt, Eric E., additional, Mani, D.R., additional, Ding, Li, additional, Iavarone, Antonio, additional, Wiznerowicz, Maciej, additional, Schürer, Stephan, additional, Chen, Xi S., additional, Heath, Allison P., additional, Rokita, Jo Lynne, additional, Nesvizhskii, Alexey I., additional, Fenyö, David, additional, Rodland, Karin D., additional, Liu, Tao, additional, Gygi, Steven P., additional, Paulovich, Amanda G., additional, Resnick, Adam C., additional, Storm, Phillip B., additional, Rood, Brian R., additional, Wang, Pei, additional, Francis, Alicia, additional, Morgan, Allison M., additional, Waanders, Angela J., additional, Viaene, Angela N., additional, Buccoliero, Anna Maria, additional, Chinnaiyan, Arul M., additional, Leonard, Carina A., additional, Kline, Cassie N., additional, Caporalini, Chiara, additional, Kinsinger, Christopher R., additional, Li, Chunde, additional, Kram, David E., additional, Hanson, Derek, additional, Appert, Elizabeth, additional, Kawaler, Emily A., additional, Raabe, Eric H., additional, Jackson, Eric M., additional, Greenfield, Jeffrey P., additional, Stone, Gabrielle S., additional, Getz, Gad, additional, Grant, Gerald, additional, Teo, Guo Ci, additional, Pollack, Ian F., additional, Cain, Jason E., additional, Foster, Jessica B., additional, Phillips, Joanna J., additional, Palma, July E., additional, Ketchum, Karen A., additional, Ruggles, Kelly V., additional, Blumenberg, Lili, additional, Cornwell, Macintosh, additional, Sarmady, Mahdi, additional, Domagalski, Marcin J., additional, Cieślik, Marcin P., additional, Santi, Mariarita, additional, Li, Marilyn M., additional, Ellis, Matthew J., additional, Wyczalkowski, Matthew A., additional, Connors, Meghan, additional, Scagnet, Mirko, additional, Gupta, Nalin, additional, Edwards, Nathan J., additional, Vitanza, Nicholas A., additional, Vaske, Olena M., additional, Becher, Oren, additional, McGarvey, Peter B., additional, Firestein, Ron, additional, Mueller, Sabine, additional, Winebrake, Samuel G., additional, Dhanasekaran, Saravana Mohan, additional, Cai, Shuang, additional, Partap, Sonia, additional, Patton, Tatiana, additional, Le, Toan, additional, Lorentzen, Travis D., additional, Liu, Wenke, additional, and Bocik, William E., additional

Published
2020
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49. Deletions in 16q24.2 are associated with autism spectrum disorder, intellectual disability and congenital renal malformation

Author

Handrigan, Gregory Ryan, Chitayat, David, Lionel, Anath C, Pinsk, Maury, Vaags, Andrea K, Marshall, Christian R, Dyack, Sarah, Escobar, Luis F, Fernandez, Bridget A, Stegman, Joseph C, Rosenfeld, Jill A, Shaffer, Lisa G, Goodenberger, McKinsey, Hodge, Jennelle C, Cain, Jason E, Babul-Hirji, Riyana, Stavropoulos, Dimitri J, Yiu, Verna, Scherer, Stephen W, and Rosenblum, Norman D

Published
2013
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