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1. Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment

Author

Keller, Michael D., Hanley, Patrick J., Chi, Yueh-Yun, Aguayo-Hiraldo, Paibel, Dvorak, Christopher C., Verneris, Michael R., Kohn, Donald B., Pai, Sung-Yun, Dávila Saldaña, Blachy J., Hanisch, Benjamin, Quigg, Troy C., Adams, Roberta H., Dahlberg, Ann, Chandrakasan, Shanmuganathan, Hasan, Hasibul, Malvar, Jemily, Jensen-Wachspress, Mariah A., Lazarski, Christopher A., Sani, Gelina, Idso, John M., Lang, Haili, Chansky, Pamela, McCann, Chase D., Tanna, Jay, Abraham, Allistair A., Webb, Jennifer L., Shibli, Abeer, Keating, Amy K., Satwani, Prakash, Muranski, Pawel, Hall, Erin, Eckrich, Michael J., Shereck, Evan, Miller, Holly, Mamcarz, Ewelina, Agarwal, Rajni, De Oliveira, Satiro N., Vander Lugt, Mark T., Ebens, Christen L., Aquino, Victor M., Bednarski, Jeffrey J., Chu, Julia, Parikh, Suhag, Whangbo, Jennifer, Lionakis, Michail, Zambidis, Elias T., Gourdine, Elizabeth, Bollard, Catherine M., and Pulsipher, Michael A.

Published
2024
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2. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study

Author

Grunebaum, Eyal, Arnold, Danielle E., Logan, Brent, Parikh, Suhag, Marsh, Rebecca A., Griffith, Linda M., Mallhi, Kanwaldeep, Chellapandian, Deepak, Lim, Stephanie Si, Deal, Christin L., Kapoor, Neena, Murguía-Favela, Luis, Falcone, Emilia Liana, Prasad, Vinod K., Touzot, Fabien, Bleesing, Jack J., Chandrakasan, Shanmuganathan, Heimall, Jennifer R., Bednarski, Jeffrey J., Broglie, Larisa A., Chong, Hey Jin, Kapadia, Malika, Prockop, Susan, Dávila Saldaña, Blachy J., Schaefer, Edo, Bauchat, Andrea L., Teira, Pierre, Chandra, Sharat, Parta, Mark, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer M., Pulsipher, Michael A., Torgerson, Troy R., Malech, Harry L., Kang, Elizabeth M., and Leiding, Jennifer W.

Published
2024
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5. A Case of Severe Combined Immunodeficiency Missed by Newborn Screening

Author

Kitcharoensakkul, Maleewan, Aluri, Jahnavi, Elsharkawi, Ibrahim, Steed, Ashley, Putnam, Christopher D, Swayampakula, Anil K, Cooper, Megan A, and Bednarski, Jeffrey J

Subjects
Biomedical and Clinical Sciences, Immunology, Humans, Infant, Infant, Newborn, Male, Neonatal Screening, Severe Combined Immunodeficiency
Published
2021

6. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

Author

Eissa, Hesham, Thakar, Monica S., Shah, Ami J., Logan, Brent R., Griffith, Linda M., Dong, Huaying, Parrott, Roberta E., O’Reilly, Richard J., Dara, Jasmeen, Kapoor, Neena, Forbes Satter, Lisa, Chandra, Sharat, Kapadia, Malika, Chandrakasan, Shanmuganathan, Knutsen, Alan, Jyonouchi, Soma C., Molinari, Lyndsay, Rayes, Ahmad, Ebens, Christen L., Teira, Pierre, Dávila Saldaña, Blachy J., Burroughs, Lauri M., Chaudhury, Sonali, Chellapandian, Deepak, Gillio, Alfred P., Goldman, Fredrick, Malech, Harry L., DeSantes, Kenneth, Cuvelier, Geoff D.E., Rozmus, Jacob, Quinones, Ralph, Yu, Lolie C., Broglie, Larisa, Aquino, Victor, Shereck, Evan, Moore, Theodore B., Vander Lugt, Mark T., Mousallem, Talal I., Oved, Joeseph H., Dorsey, Morna, Abdel-Azim, Hisham, Martinez, Caridad, Bleesing, Jacob H., Prockop, Susan, Kohn, Donald B., Bednarski, Jeffrey J., Leiding, Jennifer, Marsh, Rebecca A., Torgerson, Troy, Notarangelo, Luigi D., Pai, Sung-Yun, Pulsipher, Michael A., Puck, Jennifer M., Dvorak, Christopher C., Haddad, Elie, Buckley, Rebecca H., Cowan, Morton J., and Heimall, Jennifer

Published
2024
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7. Immunodeficiency and bone marrow failure with mosaic and germline TLR8 gain of function

Author

Aluri, Jahnavi, Bach, Alicia, Kaviany, Saara, Chiquetto Paracatu, Luana, Kitcharoensakkul, Maleewan, Walkiewicz, Magdalena A, Putnam, Christopher D, Shinawi, Marwan, Saucier, Nermina, Rizzi, Elise M, Harmon, Michael T, Keppel, Molly P, Ritter, Michelle, Similuk, Morgan, Kulm, Elaine, Joyce, Michael, de Jesus, Adriana A, Goldbach-Mansky, Raphaela, Lee, Yi-Shan, Cella, Marina, Kendall, Peggy L, Dinauer, Mary C, Bednarski, Jeffrey J, Bemrich-Stolz, Christina, Canna, Scott W, Abraham, Shirley M, Demczko, Matthew M, Powell, Jonathan, Jones, Stacie M, Scurlock, Amy M, De Ravin, Suk See, Bleesing, Jack J, Connelly, James A, Rao, V Koneti, Schuettpelz, Laura G, and Cooper, Megan A

Subjects
Stem Cell Research, Clinical Research, Genetics, Transplantation, Stem Cell Research - Nonembryonic - Human, Hematology, 2.1 Biological and endogenous factors, Aetiology, Underpinning research, 1.1 Normal biological development and functioning, Inflammatory and immune system, Good Health and Well Being, Adolescent, Adult, B-Lymphocytes, Bone Marrow Failure Disorders, Cell Differentiation, Child, Child, Preschool, Cytokines, Female, Follow-Up Studies, Gain of Function Mutation, Humans, Immunologic Deficiency Syndromes, Infant, Inflammation, Lymphocyte Activation, Male, Mosaicism, Pancytopenia, Pedigree, Prognosis, T-Lymphocytes, Toll-Like Receptor 8, Young Adult, Cardiorespiratory Medicine and Haematology, Clinical Sciences, Paediatrics and Reproductive Medicine, Immunology
Abstract

Inborn errors of immunity (IEI) are a genetically heterogeneous group of disorders with a broad clinical spectrum. Identification of molecular and functional bases of these disorders is important for diagnosis, treatment, and an understanding of the human immune response. We identified 6 unrelated males with neutropenia, infections, lymphoproliferation, humoral immune defects, and in some cases bone marrow failure associated with 3 different variants in the X-linked gene TLR8, encoding the endosomal Toll-like receptor 8 (TLR8). Interestingly, 5 patients had somatic variants in TLR8 with

Published
2021

8. Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Author

Leiding, Jennifer W., Arnold, Danielle E., Parikh, Suhag, Logan, Brent, Marsh, Rebecca A., Griffith, Linda M., Wu, Ruizhe, Kidd, Sharon, Mallhi, Kanwaldeep, Chellapandian, Deepak, Si Lim, Stephanie J., Grunebaum, Eyal, Falcone, E. Liana, Murguia-Favela, Luis, Grossman, Debbi, Prasad, Vinod K., Heimall, Jennifer R., Touzot, Fabien, Burroughs, Lauri M., Bleesing, Jack, Kapoor, Neena, Dara, Jasmeen, Williams, Olatundun, Kapadia, Malika, Oshrine, Benjamin R., Bednarski, Jeffrey J., Rayes, Ahmad, Chong, Hey, Cuvelier, Geoffrey D. E., Forbes Satter, Lisa R., Martinez, Caridad, Vander Lugt, Mark T., Yu, Lolie C., Chandrakasan, Shanmuganathan, Joshi, Avni, Prockop, Susan E., Dávila Saldaña, Blachy J., Aquino, Victor, Broglie, Larisa A., Ebens, Christen L., Madden, Lisa M., DeSantes, Kenneth, Milner, Jordan, Rangarajan, Hemalatha G., Shah, Ami J., Gillio, Alfred P., Knutsen, Alan P., Miller, Holly K., Moore, Theodore B., Graham, Pamela, Bauchat, Andrea, Bunin, Nancy J., Teira, Pierre, Petrovic, Aleksandra, Chandra, Sharat, Abdel-Azim, Hisham, Dorsey, Morna J., Birbrayer, Olga, Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Kohn, Donald B., Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer M., Pulsipher, Michael A., Torgerson, Troy R., Malech, Harry L., and Kang, Elizabeth M.

Published
2023
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9. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers.

Author

Dorsey, Morna J, Wright, Nicola AM, Chaimowitz, Natalia S, Dávila Saldaña, Blachy J, Miller, Holly, Keller, Michael D, Thakar, Monica S, Shah, Ami J, Abu-Arja, Rolla, Andolina, Jeffrey, Aquino, Victor, Barnum, JL, Bednarski, Jeffrey J, Bhatia, Monica, Bonilla, Francisco A, Butte, Manish J, Bunin, Nancy J, Chandra, Sharat, Chaudhury, Sonali, Chen, Karin, Chong, Hey, Cuvelier, Geoffrey DE, Dalal, Jignesh, DeFelice, Magee L, DeSantes, Kenneth B, Forbes, Lisa R, Gillio, Alfred, Goldman, Fred, Joshi, Avni Y, Kapoor, Neena, Knutsen, Alan P, Kobrynski, Lisa, Lieberman, Jay A, Leiding, Jennifer W, Oshrine, Benjamin, Patel, Kiran P, Prockop, Susan, Quigg, Troy C, Quinones, Ralph, Schultz, Kirk R, Seroogy, Christine, Shyr, David, Siegel, Subhadra, Smith, Angela R, Torgerson, Troy R, Vander Lugt, Mark T, Yu, Lolie C, Cowan, Morton J, Buckley, Rebecca H, Dvorak, Christopher C, Griffith, Linda M, Haddad, Elie, Kohn, Donald B, Logan, Brent, Notarangelo, Luigi D, Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael A, and Heimall, Jennifer

Subjects
Humans, Severe Combined Immunodeficiency, Disease Susceptibility, Neonatal Screening, Prognosis, Antibiotic Prophylaxis, Hematopoietic Stem Cell Transplantation, Age of Onset, Infection Control, Infant, Infant, Newborn, Disease Management, Female, Male, Public Health Surveillance, Time-to-Treatment, Surveys and Questionnaires, Clinical Decision-Making, Infections, hematopoietic stem cell transplant, newborn screening, primary immunodeficiency, prophylaxis, severe combined immunodeficiency, Stem Cell Research, Perinatal Period - Conditions Originating in Perinatal Period, Pediatric, Prevention, Regenerative Medicine, Transplantation, Infant Mortality, Health Services, Clinical Research, Infection, Immunology
Abstract

PurposeThe Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children with severe combined immunodeficiency (SCID) in a prospective natural history study of hematopoietic stem cell transplant (HSCT) outcomes over the last decade. Despite newborn screening (NBS) for SCID, infections occurred prior to HSCT. This study's objectives were to define the types and timing of infection prior to HSCT in patients diagnosed via NBS or by family history (FH) and to understand the breadth of strategies employed at PIDTC centers for infection prevention.MethodsWe analyzed retrospective data on infections and pre-transplant management in patients with SCID diagnosed by NBS and/or FH and treated with HSCT between 2010 and 2014. PIDTC centers were surveyed in 2018 to understand their practices and protocols for pre-HSCT management.ResultsInfections were more common in patients diagnosed via NBS (55%) versus those diagnosed via FH (19%) (p = 0.012). Outpatient versus inpatient management did not impact infections (47% vs 35%, respectively; p = 0.423). There was no consensus among PIDTC survey respondents as to the best setting (inpatient vs outpatient) for pre-HSCT management. While isolation practices varied, immunoglobulin replacement and antimicrobial prophylaxis were more uniformly implemented.ConclusionInfants with SCID diagnosed due to FH had lower rates of infection and proceeded to HSCT more quickly than did those diagnosed via NBS. Pre-HSCT management practices were highly variable between centers, although uses of prophylaxis and immunoglobulin support were more consistent. This study demonstrates a critical need for development of evidence-based guidelines for the pre-HSCT management of infants with SCID following an abnormal NBS.Trial registrationNCT01186913.

Published
2021

10. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium

Author

Thakar, Monica S, Logan, Brent R, Puck, Jennifer M, Dunn, Elizabeth A, Buckley, Rebecca H, Cowan, Morton J, O'Reilly, Richard J, Kapoor, Neena, Satter, Lisa Forbes, Pai, Sung-Yun, Heimall, Jennifer, Chandra, Sharat, Ebens, Christen L, Chellapandian, Deepak, Williams, Olatundun, Burroughs, Lauri M, Saldana, Blachy Davila, Rayes, Ahmad, Madden, Lisa M, Chandrakasan, Shanmuganathan, Bednarski, Jeffrey J, II, DeSantes, Kenneth B, Cuvelier, Geoffrey D E, Teira, Pierre, Gillio, Alfred P, Eissa, Hesham, Knutsen, Alan P, Goldman, Frederick D, Aquino, Victor M, Shereck, Evan B, Moore, Theodore B, Caywood, Emi H, Lugt, Mark T Vander, Rozmus, Jacob, Broglie, Larisa, Yu, Lolie C, Shah, Ami J, Andolina, Jeffrey R, Liu, Xuerong, Parrott, Roberta E, Dara, Jasmeen, Prockop, Susan, Martinez, Caridad A, Kapadia, Malika, Jyonouchi, Soma C, Sullivan, Kathleen E, Bleesing, Jack J, Chaudhury, Sonali, Petrovic, Aleksandra, Keller, Michael D, Quigg, Troy C, Parikh, Suhag, Shenoy, Shalini, Seroogy, Christine, Rubin, Tamar, Decaluwe, Hélène, Routes, John M, Torgerson, Troy R, Leiding, Jennifer W, Pulsipher, Michael A, Kohn, Donald B, Griffith, Linda M, Haddad, Elie, Dvorak, Christopher C, and Notarangelo, Luigi D

Published
2023
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11. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey.

Author

Chan, Alice Y, Leiding, Jennifer W, Liu, Xuerong, Logan, Brent R, Burroughs, Lauri M, Allenspach, Eric J, Skoda-Smith, Suzanne, Uzel, Gulbu, Notarangelo, Luigi D, Slatter, Mary, Gennery, Andrew R, Smith, Angela R, Pai, Sung-Yun, Jordan, Michael B, Marsh, Rebecca A, Cowan, Morton J, Dvorak, Christopher C, Craddock, John A, Prockop, Susan E, Chandrakasan, Shanmuganathan, Kapoor, Neena, Buckley, Rebecca H, Parikh, Suhag, Chellapandian, Deepak, Oshrine, Benjamin R, Bednarski, Jeffrey J, Cooper, Megan A, Shenoy, Shalini, Davila Saldana, Blachy J, Forbes, Lisa R, Martinez, Caridad, Haddad, Elie, Shyr, David C, Chen, Karin, Sullivan, Kathleen E, Heimall, Jennifer, Wright, Nicola, Bhatia, Monica, Cuvelier, Geoffrey DE, Goldman, Frederick D, Meyts, Isabelle, Miller, Holly K, Seidel, Markus G, Vander Lugt, Mark T, Bacchetta, Rosa, Weinacht, Katja G, Andolina, Jeffrey R, Caywood, Emi, Chong, Hey, de la Morena, Maria Teresa, Aquino, Victor M, Shereck, Evan, Walter, Jolan E, Dorsey, Morna J, Seroogy, Christine M, Griffith, Linda M, Kohn, Donald B, Puck, Jennifer M, Pulsipher, Michael A, and Torgerson, Troy R

Subjects
Animals, Humans, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Adolescent, Adult, Middle Aged, Child, Child, Preschool, Infant, T-Lymphocytes, Regulatory, Young Adult, Surveys and Questionnaires, Primary Immunodeficiency Diseases, autoimmunity, genetics, hematopoietic cell transplant, immune dysregulation, primary immune deficiencies, Clinical Research, Rare Diseases, Transplantation, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Inflammatory and immune system, Immunology, Medical Microbiology
Abstract

Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated with diminished and exaggerated immune responses. Management of these patients is complicated; oftentimes immunosuppressive therapies are insufficient, and patients may require hematopoietic cell transplant (HCT) for treatment. Analysis of HCT data in PIRD patients have previously focused on a single gene defect. This study surveyed transplanted patients with a phenotypic clinical picture consistent with PIRD treated in 33 Primary Immune Deficiency Treatment Consortium centers and European centers. Our data showed that PIRD patients often had immunodeficient and autoimmune features affecting multiple organ systems. Transplantation resulted in resolution of disease manifestations in more than half of the patients with an overall 5-years survival of 67%. This study, the first to encompass disorders across the PIRD spectrum, highlights the need for further research in PIRD management.

Published
2020

12. The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions

Author

Dvorak, Christopher C., Haddad, Elie, Heimall, Jennifer, Dunn, Elizabeth, Cowan, Morton J., Pai, Sung-Yun, Kapoor, Neena, Satter, Lisa Forbes, Buckley, Rebecca H., O’Reilly, Richard J., Chandra, Sharat, Bednarski, Jeffrey J., Williams, Olatundun, Rayes, Ahmad, Moore, Theodore B., Ebens, Christen L., Davila Saldana, Blachy J., Petrovic, Aleksandra, Chellapandian, Deepak, Cuvelier, Geoffrey D.E., Vander Lugt, Mark T., Caywood, Emi H., Chandrakasan, Shanmuganathan, Eissa, Hesham, Goldman, Frederick D., Shereck, Evan, Aquino, Victor M., Desantes, Kenneth B., Madden, Lisa M., Miller, Holly K., Yu, Lolie, Broglie, Larisa, Gillio, Alfred, Shah, Ami J., Knutsen, Alan P., Andolina, Jeffrey P., Joshi, Avni Y., Szabolcs, Paul, Kapadia, Malika, Martinez, Caridad A., Parrot, Roberta E., Sullivan, Kathleen E., Prockop, Susan E., Abraham, Roshini S., Thakar, Monica S., Leiding, Jennifer W., Kohn, Donald B., Pulsipher, Michael A., Griffith, Linda M., Notarangelo, Luigi D., and Puck, Jennifer M.

Published
2023
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14. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Author

Cuvelier, Geoffrey D.E., Logan, Brent R., Prockop, Susan E., Buckley, Rebecca H., Kuo, Caroline Y., Griffith, Linda M., Liu, Xuerong, Yip, Alison, Hershfield, Michael S., Ayoub, Paul G., Moore, Theodore B., Dorsey, Morna J., O'Reilly, Richard J., Kapoor, Neena, Pai, Sung-Yun, Kapadia, Malika, Ebens, Christen L., Forbes Satter, Lisa R., Burroughs, Lauri M., Petrovic, Aleksandra, Chellapandian, Deepak, Heimall, Jennifer, Shyr, David C., Rayes, Ahmad, Bednarski, Jeffrey J., Chandra, Sharat, Chandrakasan, Shanmuganathan, Gillio, Alfred P., Madden, Lisa, Quigg, Troy C., Caywood, Emi H., Dávila Saldaña, Blachy J., DeSantes, Kenneth, Eissa, Hesham, Goldman, Frederick D., Rozmus, Jacob, Shah, Ami J., Vander Lugt, Mark T., Thakar, Monica S., Parrott, Roberta E., Martinez, Caridad, Leiding, Jennifer W., Torgerson, Troy R., Pulsipher, Michael A., Notarangelo, Luigi D., Cowan, Morton J., Dvorak, Christopher C., Haddad, Elie, Puck, Jennifer M., and Kohn, Donald B.

Published
2022
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15. Donor memory-like NK cells persist and induce remissions in pediatric patients with relapsed AML after transplant

Author

Bednarski, Jeffrey J., Zimmerman, Clare, Berrien-Elliott, Melissa M., Foltz, Jennifer A., Becker-Hapak, Michelle, Neal, Carly C., Foster, Mark, Schappe, Timothy, McClain, Ethan, Pence, Patrick P., Desai, Sweta, Kersting-Schadek, Samantha, Wong, Pamela, Russler-Germain, David A., Fisk, Bryan, Lie, Wen-Rong, Eisele, Jeremy, Hyde, Stephanie, Bhatt, Sima T., Griffith, Obi L., Griffith, Malachi, Petti, Allegra A., Cashen, Amanda F., and Fehniger, Todd A.

Published
2022
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17. Outcomes Following Matched Sibling Donor Transplant for Severe Combined Immunodeficiency: A Report from the Pidtc

Author

Rayes, Ahmad, primary, Logan, Brent, additional, Liu, Xuerong, additional, Dara, Jasmeen, additional, Buckley, Rebecca H., additional, Oved, Joseph H., additional, Kapoor, Neena, additional, Kapadia, Malika, additional, Chandra, Sharat, additional, Martinez, Caridad, additional, Bunin, Nancy J., additional, Chandrakasan, Shanmuganathan, additional, Burroughs, Lauri M., additional, Bednarski, Jeffrey J, additional, Haines, Hilary, additional, Cuvelier, Geoff D.E., additional, Eissa, Hesham, additional, Talano, Julie A, additional, Saldana, Blachy J, additional, Ebens, Christen L., additional, Chaudhury, Sonali, additional, Shereck, Evan, additional, Aquino, Victor, additional, Knutsen, Alan P., additional, Alexander, Jessie L, additional, Gillio, Alfred P., additional, Chellapandian, Deepak, additional, Shah, Ami J., additional, Miller, Holly K., additional, Lugt, Mark T. Vander, additional, DeSantes, Kenneth, additional, Dorsey, Morna, additional, Parrott, Roberta E, additional, O'Reilly, Richard J., additional, Aguayo-Hiraldo, Paibel, additional, Torgerson, Troy, additional, Leiding, Jennifer W., additional, Marsh, Rebecca A., additional, Griffith, Linda M., additional, Pulsipher, Mike A., additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Cowan, Morton J., additional, Puck, Jennifer, additional, Heimall, Jennifer R., additional, Haddad, Elie, additional, Pai, Sung-Yun, additional, and Dvorak, Christopher C., additional

Published
2024
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18. Donor-Derived Memory-like NK Cells for the Treatment of Children and Young Adults with Relapsed AML Following Allo-HCT

Author

Pfeiffer, Thomas, primary, Zimmerman, Clare, additional, Berrien-Elliott, Melissa M, additional, Foltz, Jennifer, additional, Becker-Hapak, Michelle, additional, Neal, Carly, additional, Foster, Mark, additional, Schappe, Timothy, additional, McClain, Ethan, additional, Pence, Patrick, additional, Desai, Sweta, additional, Wong, Pamela, additional, Russler-Germain, David, additional, Fisk, Bryan, additional, Wen-Rong, Lie, additional, Eisele, Jeremy, additional, Hyde, Stephanie, additional, Bhatt, Sima, additional, Griffith, Obi, additional, Griffith, Malachi, additional, Rueve, Joseph, additional, Pureti, Sushanth, additional, Cashen, Amanda, additional, Bednarski, Jeffrey J, additional, and Fehniger, Todd A, additional

Published
2024
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22. A Novel Secreted Protein, MYR1, Is Central to Toxoplasma’s Manipulation of Host Cells

Author

Franco, Magdalena, Panas, Michael W, Marino, Nicole D, Lee, Mei-Chong Wendy, Buchholz, Kerry R, Kelly, Felice D, Bednarski, Jeffrey J, Sleckman, Barry P, Pourmand, Nader, and Boothroyd, John C

Subjects
Biochemistry and Cell Biology, Biological Sciences, Vaccine Related, Emerging Infectious Diseases, Infectious Diseases, Biotechnology, Prevention, Foodborne Illness, Genetics, Biodefense, 2.1 Biological and endogenous factors, 2.2 Factors relating to the physical environment, Aetiology, Infection, Generic health relevance, Animals, Gene Deletion, Host-Pathogen Interactions, Macrophages, Mice, Protozoan Proteins, Toxoplasma, Toxoplasmosis, Animal, Virulence Factors, Microbiology, Biochemistry and cell biology, Medical microbiology
Abstract

UnlabelledThe intracellular protozoan Toxoplasma gondii dramatically reprograms the transcriptome of host cells it infects, including substantially up-regulating the host oncogene c-myc. By applying a flow cytometry-based selection to infected mouse cells expressing green fluorescent protein fused to c-Myc (c-Myc-GFP), we isolated mutant tachyzoites defective in this host c-Myc up-regulation. Whole-genome sequencing of three such mutants led to the identification of MYR1 (Myc regulation 1; TGGT1_254470) as essential for c-Myc induction. MYR1 is a secreted protein that requires TgASP5 to be cleaved into two stable portions, both of which are ultimately found within the parasitophorous vacuole and at the parasitophorous vacuole membrane. Deletion of MYR1 revealed that in addition to its requirement for c-Myc up-regulation, the MYR1 protein is needed for the ability of Toxoplasma tachyzoites to modulate several other important host pathways, including those mediated by the dense granule effectors GRA16 and GRA24. This result, combined with its location at the parasitophorous vacuole membrane, suggested that MYR1 might be a component of the machinery that translocates Toxoplasma effectors from the parasitophorous vacuole into the host cytosol. Support for this possibility was obtained by showing that transit of GRA24 to the host nucleus is indeed MYR1-dependent. As predicted by this pleiotropic phenotype, parasites deficient in MYR1 were found to be severely attenuated in a mouse model of infection. We conclude, therefore, that MYR1 is a novel protein that plays a critical role in how Toxoplasma delivers effector proteins to the infected host cell and that this is crucial to virulence.ImportanceToxoplasma gondii is an important human pathogen and a model for the study of intracellular parasitism. Infection of the host cell with Toxoplasma tachyzoites involves the introduction of protein effectors, including many that are initially secreted into the parasitophorous vacuole but must ultimately translocate to the host cell cytosol to function. The work reported here identified a novel protein that is required for this translocation. These results give new insight into a very unusual cell biology process as well as providing a potential handle on a pathway that is necessary for virulence and, therefore, a new potential target for chemotherapy.

Published
2016

24. MYSM1 attenuates DNA damage signals triggered by physiologic and genotoxic DNA breaks

Author

Mathias, Brendan, primary, O’Leary, David, additional, Saucier, Nermina, additional, Ahmad, Faiz, additional, White, Lynn S., additional, Russell, Le’Mark, additional, Shinawi, Marwan, additional, Smith, Matthew J., additional, Abraham, Roshini S., additional, Cooper, Megan A., additional, Kitcharoensakkul, Maleewan, additional, Green, Abby M., additional, and Bednarski, Jeffrey J., additional

Published
2023
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25. The SMC5/6 complex is required for maintenance of genome integrity upon APOBEC3A-mediated replication stress

Author

O'Leary, David R, primary, Hansen, Ava R, additional, Fingerman, Dylan F, additional, Tran, Thi A, additional, Harris, Brooke R, additional, Hayer, Katharina E, additional, DeWeerd, Rachel A, additional, Tennekoon, Mithila, additional, Meroni, Alice, additional, Szeto, Julia, additional, Weitzman, Matthew D, additional, Bednarski, Jeffrey J, additional, Shalem, Ophir, additional, Vindigni, Alessandro, additional, and Green, Abby Margaret, additional

Published
2023
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26. Neuroinflammatory Disease as an Isolated Manifestation of Hemophagocytic Lymphohistiocytosis

Author

Blincoe, Annaliesse, Heeg, Maximilian, Campbell, Patrick K., Hines, Melissa, Khojah, Amer, Klein-Gitelman, Marisa, Talano, Julie-An, Speckmann, Carsten, Touzot, Fabien, Lankester, Arjan, Legger, Geertje E., Rivière, Jacques G., Garcia-Prat, Marina, Alonso, Laura, Putti, Maria C., Lehmberg, Kai, Maier, Sarah, El Chazli, Yasmine, Elmaksoud, Marwa Abd, Astigarraga, Itziar, Kurjane, Natalja, Bulina, Inita, Kenina, Viktorija, Bryceson, Yenan, Rascon, Jelena, Lortie, Anne, Goldstein, Gal, Booth, Claire, Worth, Austen, Wassmer, Evangeline, Schmitt, Erica G., Warren, Julia T., Bednarski, Jeffrey J., Ali, Salah, Chiang, Kuang-Yueh, Krueger, Joerg, Henry, Michael M., Holland, Steven M., Marsh, Rebecca A., Ehl, Stephan, and Haddad, Elie

Published
2020
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27. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers

Author

Dorsey, Morna, Wright, Nicola A. M., Chaimowitz, Natalia S., Dávila Saldaña, Blachy J., Miller, Holly, Keller, Michael D., Thakar, Monica S., Shah, Ami J., Abu-Arja, Rolla, Andolina, Jeffrey, Aquino, Victor, Barnum, J. L., Bednarski, Jeffrey J., Bhatia, Monica, Bonilla, Francisco A., Butte, Manish J., Bunin, Nancy J., Burroughs, Lauri M., Chandra, Sharat, Chaudhury, Sonali, Chen, Karin, Chong, Hey, Cuvelier, Geoff, Dalal, Jignesh, DeFelice, Magee L., DeSantes, Kenneth B., Forbes, Lisa R., Gillio, Alfred, Goldman, Fred, Joshi, Avni Y., Kapoor, Neena, Knutsen, Alan P., Kobrynski, Lisa, Lieberman, Jay A., Leiding, Jennifer W., Oshrine, Benjamin, Patel, Kiran P., Prockop, Susan, Quigg, Troy C., Quinones, Ralph, Schultz, Kirk R., Seroogy, Christine, Shyr, David, Siegel, Subhadra, Smith, Angela R., Torgerson, Troy R., Vander Lugt, Mark T., Yu, Lolie C., Cowan, Morton J., Buckley, Rebecca H., Dvorak, Christopher C., Griffith, Linda M., Haddad, Elie, Kohn, Donald B., Logan, Brent, Notarangelo, Luigi D., Pai, Sung-Yun, Puck, Jennifer, Pulsipher, Michael A., and Heimall, Jennifer

Published
2021
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29. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

Author

Eissa, Hesham, primary, Thakar, Monica S., additional, Shah, Ami J., additional, Logan, Brent R., additional, Griffith, Linda M., additional, Dong, Huaying, additional, Parrott, Roberta E., additional, O’Reilly, Richard J., additional, Dara, Jasmeen, additional, Kapoor, Neena, additional, Forbes Satter, Lisa, additional, Chandra, Sharat, additional, Kapadia, Malika, additional, Chandrakasan, Shanmuganathan, additional, Knutsen, Alan, additional, Jyonouchi, Soma C., additional, Molinari, Lyndsay, additional, Rayes, Ahmad, additional, Ebens, Christen L., additional, Teira, Pierre, additional, Dávila Saldaña, Blachy J., additional, Burroughs, Lauri M., additional, Chaudhury, Sonali, additional, Chellapandian, Deepak, additional, Gillio, Alfred P., additional, Goldman, Fredrick, additional, Malech, Harry L., additional, DeSantes, Kenneth, additional, Cuvelier, Geoff D.E., additional, Rozmus, Jacob, additional, Quinones, Ralph, additional, Yu, Lolie C., additional, Broglie, Larisa, additional, Aquino, Victor, additional, Shereck, Evan, additional, Moore, Theodore B., additional, Vander Lugt, Mark T., additional, Mousallem, Talal I., additional, Oved, Joeseph H., additional, Dorsey, Morna, additional, Abdel-Azim, Hisham, additional, Martinez, Caridad, additional, Bleesing, Jacob H., additional, Prockop, Susan, additional, Kohn, Donald B., additional, Bednarski, Jeffrey J., additional, Leiding, Jennifer, additional, Marsh, Rebecca A., additional, Torgerson, Troy, additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Pulsipher, Michael A., additional, Puck, Jennifer M., additional, Dvorak, Christopher C., additional, Haddad, Elie, additional, Buckley, Rebecca H., additional, Cowan, Morton J., additional, and Heimall, Jennifer, additional

Published
2023
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30. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery

Author

Haddad, Elie, Logan, Brent R., Griffith, Linda M., Buckley, Rebecca H., Parrott, Roberta E., Prockop, Susan E., Small, Trudy N., Chaisson, Jessica, Dvorak, Christopher C., Murnane, Megan, Kapoor, Neena, Abdel-Azim, Hisham, Hanson, Imelda C., Martinez, Caridad, Bleesing, Jack J.H., Chandra, Sharat, Smith, Angela R., Cavanaugh, Matthew E., Jyonouchi, Soma, Sullivan, Kathleen E., Burroughs, Lauri, Skoda-Smith, Suzanne, Haight, Ann E., Tumlin, Audrey G., Quigg, Troy C., Taylor, Candace, Dávila Saldaña, Blachy J., Keller, Michael D., Seroogy, Christine M., Desantes, Kenneth B., Petrovic, Aleksandra, Leiding, Jennifer W., Shyr, David C., Decaluwe, Hélène, Teira, Pierre, Gillio, Alfred P., Knutsen, Alan P., Moore, Theodore B., Kletzel, Morris, Craddock, John A., Aquino, Victor, Davis, Jeffrey H., Yu, Lolie C., Cuvelier, Geoffrey D.E., Bednarski, Jeffrey J., Goldman, Frederick D., Kang, Elizabeth M., Shereck, Evan, Porteus, Matthew H., Connelly, James A., Fleisher, Thomas A., Malech, Harry L., Shearer, William T., Szabolcs, Paul, Thakar, Monica S., Vander Lugt, Mark T., Heimall, Jennifer, Yin, Ziyan, Pulsipher, Michael A., Pai, Sung-Yun, Kohn, Donald B., Puck, Jennifer M., Cowan, Morton J., O'Reilly, Richard J., and Notarangelo, Luigi D.

Published
2018
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33. Brief IL-12, IL-15, IL-18 activation drives the acquisition of two distinct memory-like NK cell states

Author

Foltz, Jennifer Ann, primary, Tran, Jennifer, additional, Fisk, Bryan, additional, Wong, Pamela, additional, Khan, Saad M., additional, Berrien-Elliott, Melissa M., additional, Becker-Hapak, Michelle, additional, Russler-Germain, David A., additional, Marsala, Lynne, additional, Bednarski, Jeffrey J., additional, Zimmerman, Clare, additional, Cubitt, Celia C., additional, Marin, Nancy D., additional, Zhou, Alice Y., additional, Jacobs, Miriam T., additional, Foster, Mark, additional, Schappe, Timothy, additional, Pence, Patrick, additional, Cashen, Amanda, additional, Griffith, Obi L., additional, Griffith, Malachi, additional, Petti, Allegra A., additional, and Fehniger, Todd A, additional

Published
2023
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35. Event Free Survival in Severe Combined Immune Deficiency (SCID) Infants after Conditioned Umbilical Cord Blood Transplantation (UCBT) Benefits from Omitting Serotherapy

Author

Martinez, Caridad, primary, Logan, Brent, additional, Liu, Xuerong, additional, Dvorak, Christopher C., additional, Madden, Lisa, additional, Molinari, Lyndsay, additional, Cowan, Morton J., additional, Pai, Sung-Yun, additional, Haddad, Elie, additional, Puck, Jennifer, additional, Kohn, Donald B., additional, Griffith, Linda M., additional, Pulsipher, Michael, additional, Leiding, Jennifer W., additional, Notarangelo, Luigi D., additional, Torgerson, Troy, additional, Marsh, Rebecca A., additional, Cuvelier, Geoff D.E., additional, Prockop, Susan, additional, Buckley, Rebecca H., additional, Kuo, Caroline Y., additional, Yip, Alison, additional, Hershfield, Michael S., additional, Parrott, Roberta E, additional, Ebens, Christen L., additional, Moore, Theodore B., additional, O’Reilly, Richard J., additional, Kapadia, Malika, additional, Kapoor, Neena, additional, Satter, Lisa Forbes, additional, Burroughs, Lauri M., additional, Petrovic, Aleksandra, additional, Thakar, Monica S., additional, Chellapandian, Deepak, additional, Heimall, Jennifer R., additional, Shyr, David C., additional, Bednarski, Jeffrey J, additional, Rayes, Ahmad, additional, Chandrakasan, Shanmuganathan, additional, Quigg, Troy C., additional, Davila, Blachy J, additional, DeSantes, Kenneth, additional, Eissa, Hesham, additional, Goldman, Frederick, additional, Rozmus, Jacob, additional, Shah, Ami J, additional, Lugt, Mark Vander, additional, Keller, Michael D., additional, Sullivan, Kathleen E., additional, Jyonouchi, Soma, additional, Seroogy, Christine, additional, Decaluwe, Helene, additional, Teira, Pierre, additional, Knutsen, Alan P., additional, Kletzel, Morris, additional, Aquino, Victor, additional, Davis, Jeffrey H, additional, and Szabolcs, Paul, additional

Published
2023
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37. Allogenic Hematopoietic Cell Transplantations Are Effective in Patients with p47phox Chronic Granulomatous Disease: A Primary Immune Deficiency Treatment Consortium Study

Author

Grunebaum, Eyal, primary, Arnold, Danielle E., additional, Logan, Brent, additional, Parikh, Suhag, additional, Marsh, Rebecca A., additional, Griffith, Linda M., additional, Mallhi, Kanwaldeep, additional, Chellapandian, Deepak, additional, Lim, Stephanie Si, additional, Deal, Christin L., additional, Murguía-Favela, Luis, additional, Mousallem, Talal I., additional, Prasad, Prof. Vinod K., additional, Teira, Pierre, additional, Touzot, Fabien, additional, Bunin, Nancy J., additional, Heimall, Jennifer R., additional, Burroughs, Lauri M., additional, Kapadia, Malika, additional, Prockop, Susan, additional, Chandra, Sharat, additional, Chandrakasan, Shanmuganathan, additional, Chaudhury, Sonali, additional, Broglie, Larisa, additional, O’Reilly, Richard J., additional, Dávila Saldaña, Blachy J., additional, Schaefer, Edo, additional, Chong, Hey, additional, Bednarski, Jeffrey J., additional, Rayes, Ahmad, additional, DeSantes, Kenneth, additional, Kohn, Donald B., additional, Notarangelo, Luigi D., additional, Pai, Sung-Yun, additional, Puck, Jennifer, additional, Torgerson, Troy, additional, Cowan, Morton J., additional, Dvorak, Christopher C., additional, Satter, Lisa Forbes, additional, Haddad, Elie, additional, Pulsipher, Michael, additional, Malech, Harry L., additional, Kang, Elizabeth M., additional, and Leiding, Jennifer W., additional

Published
2023
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43. 116 - Event Free Survival in Severe Combined Immune Deficiency (SCID) Infants after Conditioned Umbilical Cord Blood Transplantation (UCBT) Benefits from Omitting Serotherapy

Author

Martinez, Caridad, Logan, Brent, Liu, Xuerong, Dvorak, Christopher C., Madden, Lisa, Molinari, Lyndsay, Cowan, Morton J., Pai, Sung-Yun, Haddad, Elie, Puck, Jennifer, Kohn, Donald B., Griffith, Linda M., Pulsipher, Michael, Leiding, Jennifer W., Notarangelo, Luigi D., Torgerson, Troy, Marsh, Rebecca A., Cuvelier, Geoff D.E., Prockop, Susan, Buckley, Rebecca H., Kuo, Caroline Y., Yip, Alison, Hershfield, Michael S., Parrott, Roberta E, Ebens, Christen L., Moore, Theodore B., O’Reilly, Richard J., Kapadia, Malika, Kapoor, Neena, Satter, Lisa Forbes, Burroughs, Lauri M., Petrovic, Aleksandra, Thakar, Monica S., Chellapandian, Deepak, Heimall, Jennifer R., Shyr, David C., Bednarski, Jeffrey J, Rayes, Ahmad, Chandrakasan, Shanmuganathan, Quigg, Troy C., Davila, Blachy J, DeSantes, Kenneth, Eissa, Hesham, Goldman, Frederick, Rozmus, Jacob, Shah, Ami J, Lugt, Mark Vander, Keller, Michael D., Sullivan, Kathleen E., Jyonouchi, Soma, Seroogy, Christine, Decaluwe, Helene, Teira, Pierre, Knutsen, Alan P., Kletzel, Morris, Aquino, Victor, Davis, Jeffrey H, and Szabolcs, Paul

Published
2023
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48. Cytokine-Induced Memory-like NK Cells Have a Distinct Single Cell Transcriptional Profile and Persist for Months in Adult and Pediatric Leukemia Patients after Adoptive Transfer

Author

Foltz, Jennifer A., primary, Berrien-Elliott, Melissa M., additional, Russler-Germain, David A., additional, Neal, Carly C., additional, Tran, Jennifer, additional, Gang, Margery, additional, Wong, Pamela, additional, Mosior, Matthew, additional, Bednarski, Jeffrey J., additional, Zimmerman, Clare, additional, Cubitt, Celia C., additional, Marin, Nancy D., additional, Zhou, Alice Y., additional, Jacobs, Miriam T., additional, Foster, Mark, additional, Schappe, Timothy, additional, McClain, Ethan, additional, Desai, Sweta, additional, Pence, Patrick, additional, Becker-Hapak, Michelle, additional, Marsala, Lynne, additional, Griffith, Obi L., additional, Griffith, Malachi, additional, Khan, Saad M., additional, Fisk, Bryan, additional, Cashen, Amanda F., additional, Petti, Allegra A., additional, and Fehniger, Todd A., additional

Published
2021
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50. Nuclease‐independent functions of RAG1 direct distinct DNA damage responses in B cells.

Author

Johnston, Rachel, Mathias, Brendan, Crowley, Stephanie J, Schmidt, Haley A, White, Lynn S, Mosammaparast, Nima, Green, Abby M, and Bednarski, Jeffrey J

Abstract

Developing B cells generate DNA double‐stranded breaks (DSBs) to assemble immunoglobulin receptor (Ig) genes necessary for the expression of a mature B cell receptor. These physiologic DSBs are made by the RAG endonuclease, which is comprised of the RAG1 and RAG2 proteins. In pre‐B cells, RAG‐mediated DSBs activate the ATM kinase to coordinate canonical and non‐canonical DNA damage responses (DDR) that trigger DSB repair and B cell developmental signals, respectively. Whether this broad cellular response is distinctive to RAG DSBs is poorly understood. To delineate the factors that direct DDR signaling in B cells, we express a tetracycline‐inducible Cas9 nuclease in Rag1‐deficient pre‐B cells. Both RAG‐ and Cas9‐mediated DSBs at Ig genes activate canonical DDR. In contrast, RAG DSBs, but not Cas9 DSBs, induce the non‐canonical DDR‐dependent developmental program. This unique response to RAG DSBs is, in part, regulated by non‐core regions of RAG1. Thus, B cells trigger distinct cellular responses to RAG DSBs through unique properties of the RAG endonuclease that promotes activation of B cell developmental programs. Synopsis: Both Cas9‐ and RAG‐mediated DNA breaks initiate canonical DNA damage responses in pre‐B cells. RAG‐mediated DNA breaks also uniquely activate non‐canonical developmental signals through properties of the RAG1 endonuclease. Pre‐B cells induce distinct DNA damage responses based on the mechanism of DNA injury.All DNA breaks activate canonical pathways to promote repair.Only RAG‐generated DNA breaks trigger non‐canonical pathways that promote pre‐B cell survival and differentiation.The unique response to RAG‐mediated DNA breaks is, in part, regulated by non‐core regions of RAG1. [ABSTRACT FROM AUTHOR]

Published
2023
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