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947 results on '"Murry, Charles E."'

2. Modulation of FGF pathway signaling and vascular differentiation using designed oligomeric assemblies

Author

Edman, Natasha I., Phal, Ashish, Redler, Rachel L., Schlichthaerle, Thomas, Srivatsan, Sanjay R., Ehnes, Devon Duron, Etemadi, Ali, An, Seong J., Favor, Andrew, Li, Zhe, Praetorius, Florian, Gordon, Max, Vincent, Thomas, Marchiano, Silvia, Blakely, Leslie, Lin, Chuwei, Yang, Wei, Coventry, Brian, Hicks, Derrick R., Cao, Longxing, Bethel, Neville, Heine, Piper, Murray, Analisa, Gerben, Stacey, Carter, Lauren, Miranda, Marcos, Negahdari, Babak, Lee, Sangwon, Trapnell, Cole, Zheng, Ying, Murry, Charles E., Schweppe, Devin K., Freedman, Benjamin S., Stewart, Lance, Ekiert, Damian C., Schlessinger, Joseph, Shendure, Jay, Bhabha, Gira, Ruohola-Baker, Hannele, and Baker, David

Published
2024
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5. Gain-of-function cardiomyopathic mutations in RBM20 rewire splicing regulation and re-distribute ribonucleoprotein granules within processing bodies

Author

Fenix, Aidan M, Miyaoka, Yuichiro, Bertero, Alessandro, Blue, Steven M, Spindler, Matthew J, Tan, Kenneth KB, Perez-Bermejo, Juan A, Chan, Amanda H, Mayerl, Steven J, Nguyen, Trieu D, Russell, Caitlin R, Lizarraga, Paweena P, Truong, Annie, So, Po-Lin, Kulkarni, Aishwarya, Chetal, Kashish, Sathe, Shashank, Sniadecki, Nathan J, Yeo, Gene W, Murry, Charles E, Conklin, Bruce R, and Salomonis, Nathan

Subjects
Biological Sciences, Bioinformatics and Computational Biology, Rare Diseases, Heart Disease, Genetics, Cardiovascular, Aetiology, 2.1 Biological and endogenous factors, Cardiomyopathies, Cardiomyopathy, Dilated, DEAD-box RNA Helicases, DNA Helicases, Gain of Function Mutation, Gene Knockdown Techniques, Humans, Induced Pluripotent Stem Cells, Male, Mutation, Mutation, Missense, Poly-ADP-Ribose Binding Proteins, Proto-Oncogene Proteins, RNA Helicases, RNA Recognition Motif Proteins, RNA Splicing, RNA-Binding Proteins, Ribonucleoproteins
Abstract

Mutations in the cardiac splicing factor RBM20 lead to malignant dilated cardiomyopathy (DCM). To understand the mechanism of RBM20-associated DCM, we engineered isogenic iPSCs with DCM-associated missense mutations in RBM20 as well as RBM20 knockout (KO) iPSCs. iPSC-derived engineered heart tissues made from these cell lines recapitulate contractile dysfunction of RBM20-associated DCM and reveal greater dysfunction with missense mutations than KO. Analysis of RBM20 RNA binding by eCLIP reveals a gain-of-function preference of mutant RBM20 for 3' UTR sequences that are shared with amyotrophic lateral sclerosis (ALS) and processing-body associated RNA binding proteins (FUS, DDX6). Deep RNA sequencing reveals that the RBM20 R636S mutant has unique gene, splicing, polyadenylation and circular RNA defects that differ from RBM20 KO. Super-resolution microscopy verifies that mutant RBM20 maintains very limited nuclear localization potential; rather, the mutant protein associates with cytoplasmic processing bodies (DDX6) under basal conditions, and with stress granules (G3BP1) following acute stress. Taken together, our results highlight a pathogenic mechanism in cardiac disease through splicing-dependent and -independent pathways.

Published
2021

7. Author Correction: TFPa/HADHA is required for fatty acid beta-oxidation and cardiolipin re-modeling in human cardiomyocytes

Author

Miklas, Jason W, Clark, Elisa, Levy, Shiri, Detraux, Damien, Leonard, Andrea, Beussman, Kevin, Showalter, Megan R, Smith, Alec T, Hofsteen, Peter, Yang, Xiulan, Macadangdang, Jesse, Manninen, Tuula, Raftery, Daniel, Madan, Anup, Suomalainen, Anu, Kim, Deok-Ho, Murry, Charles E, Fiehn, Oliver, Sniadecki, Nathan J, Wang, Yuliang, and Ruohola-Baker, Hannele

Subjects
Engineering, Electrical Engineering
Abstract

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Published
2020

11. TFPa/HADHA is required for fatty acid beta-oxidation and cardiolipin re-modeling in human cardiomyocytes

Author

Miklas, Jason W, Clark, Elisa, Levy, Shiri, Detraux, Damien, Leonard, Andrea, Beussman, Kevin, Showalter, Megan R, Smith, Alec T, Hofsteen, Peter, Yang, Xiulan, Macadangdang, Jesse, Manninen, Tuula, Raftery, Daniel, Madan, Anup, Suomalainen, Anu, Kim, Deok-Ho, Murry, Charles E, Fiehn, Oliver, Sniadecki, Nathan J, Wang, Yuliang, and Ruohola-Baker, Hannele

Subjects
Cardiovascular, Genetics, Pediatric, 2.1 Biological and endogenous factors, Aetiology, Calcium, Cardiolipins, Cell Line, Electrophysiology, Fatty Acids, Homeodomain Proteins, Human Embryonic Stem Cells, Humans, MicroRNAs, Mitochondria, Mitochondrial Trifunctional Protein, Mitochondrial Trifunctional Protein, alpha Subunit, Myocytes, Cardiac, Oxidation-Reduction, Patch-Clamp Techniques, RNA-Seq, Tumor Suppressor Proteins
Abstract

Mitochondrial trifunctional protein deficiency, due to mutations in hydratase subunit A (HADHA), results in sudden infant death syndrome with no cure. To reveal the disease etiology, we generated stem cell-derived cardiomyocytes from HADHA-deficient hiPSCs and accelerated their maturation via an engineered microRNA maturation cocktail that upregulated the epigenetic regulator, HOPX.  Here we report, matured HADHA mutant cardiomyocytes treated with an endogenous mixture of fatty acids manifest the disease phenotype: defective calcium dynamics and repolarization kinetics which results in a pro-arrhythmic state. Single cell RNA-seq reveals a cardiomyocyte developmental intermediate, based on metabolic gene expression. This intermediate gives rise to mature-like cardiomyocytes in control cells but, mutant cells transition to a pathological state with reduced fatty acid beta-oxidation, reduced mitochondrial proton gradient, disrupted cristae structure and defective cardiolipin remodeling. This study reveals that HADHA (tri-functional protein alpha), a monolysocardiolipin acyltransferase-like enzyme, is required for fatty acid beta-oxidation and cardiolipin remodeling, essential for functional mitochondria in human cardiomyocytes.

Published
2019

13. Long-term engraftment and maturation of autologous iPSC-derived cardiomyocytes in two rhesus macaques

Author

Lin, Yongshun, primary, Sato, Noriko, additional, Hong, Sogun, additional, Nakamura, Kenta, additional, Ferrante, Elisa A., additional, Yu, Zu Xi, additional, Chen, Marcus Y., additional, Nakamura, Daisy S., additional, Yang, Xiulan, additional, Clevenger, Randall R., additional, Hunt, Timothy J., additional, Taylor, Joni L., additional, Jeffries, Kenneth R., additional, Keeran, Karen J., additional, Neidig, Lauren E., additional, Mehta, Atul, additional, Schwartzbeck, Robin, additional, Yu, Shiqin Judy, additional, Kelly, Conor, additional, Navarengom, Keron, additional, Takeda, Kazuyo, additional, Adler, Stephen S., additional, Choyke, Peter L., additional, Zou, Jizhong, additional, Murry, Charles E., additional, Boehm, Manfred, additional, and Dunbar, Cynthia E., additional

Published
2024
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15. Cardiomyocyte Regeneration

Author

Eschenhagen, Thomas, Bolli, Roberto, Braun, Thomas, Field, Loren J, Fleischmann, Bernd K, Frisén, Jonas, Giacca, Mauro, Hare, Joshua M, Houser, Steven, Lee, Richard T, Marbán, Eduardo, Martin, James F, Molkentin, Jeffery D, Murry, Charles E, Riley, Paul R, Ruiz-Lozano, Pilar, Sadek, Hesham A, Sussman, Mark A, and Hill, Joseph A

Subjects
Animals, Cardiology, Cell Proliferation, Consensus, Heart Diseases, Humans, Myocytes, Cardiac, Recovery of Function, Regeneration, Regenerative Medicine, Stem Cell Transplantation, Treatment Outcome, Cardiorespiratory Medicine and Haematology, Clinical Sciences, Public Health and Health Services, Cardiovascular System & Hematology
Published
2017

17. Genetically Encoded XTEN‐based Hydrogels with Tunable Viscoelasticity and Biodegradability for Injectable Cell Therapies

Author

Bennett, Jennifer I., primary, Boit, Mary O'Kelly, additional, Gregorio, Nicole E., additional, Zhang, Fan, additional, Kibler, Ryan D., additional, Hoye, Jack W., additional, Prado, Olivia, additional, Rapp, Peter B., additional, Murry, Charles E., additional, Stevens, Kelly R., additional, and DeForest, Cole A., additional

Published
2024
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18. Multiplexed Functional Assessments of MYH7 Variants in Human Cardiomyocytes

Author

Friedman, Clayton E., primary, Fayer, Shawn, additional, Pendyala, Sriram, additional, Chien, Wei-Ming, additional, Loiben, Alexander, additional, Tran, Linda, additional, Chao, Leslie S., additional, McKinstry, Ashley, additional, Ahmed, Dania, additional, Farris, Stephen D., additional, Stempien-Otero, April, additional, Jonlin, Erica C., additional, Murry, Charles E., additional, Starita, Lea M., additional, Fowler, Douglas M., additional, and Yang, Kai-Chun, additional

Published
2024
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20. Distilling complexity to advance cardiac tissue engineering

Author

Ogle, Brenda M, Bursac, Nenad, Domian, Ibrahim, Huang, Ngan F, Menasché, Philippe, Murry, Charles E, Pruitt, Beth, Radisic, Milica, Wu, Joseph C, Wu, Sean M, Zhang, Jianyi, Zimmermann, Wolfram-Hubertus, and Vunjak-Novakovic, Gordana

Subjects
Biomedical and Clinical Sciences, Engineering, Cardiovascular Medicine and Haematology, Biomedical Engineering, Heart Disease, Bioengineering, Regenerative Medicine, Cardiovascular, Animals, Heart Diseases, Humans, Myocardium, Myocytes, Cardiac, Tissue Engineering, Biological Sciences, Medical and Health Sciences, Medical biotechnology, Biomedical engineering
Abstract

The promise of cardiac tissue engineering is in the ability to recapitulate in vitro the functional aspects of a healthy heart and disease pathology as well as to design replacement muscle for clinical therapy. Parts of this promise have been realized; others have not. In a meeting of scientists in this field, five central challenges or "big questions" were articulated that, if addressed, could substantially advance the current state of the art in modeling heart disease and realizing heart repair.

Published
2016

21. Setting Global Standards for Stem Cell Research and Clinical Translation: The 2016 ISSCR Guidelines.

Author

Daley, George Q, Hyun, Insoo, Apperley, Jane F, Barker, Roger A, Benvenisty, Nissim, Bredenoord, Annelien L, Breuer, Christopher K, Caulfield, Timothy, Cedars, Marcelle I, Frey-Vasconcells, Joyce, Heslop, Helen E, Jin, Ying, Lee, Richard T, McCabe, Christopher, Munsie, Megan, Murry, Charles E, Piantadosi, Steven, Rao, Mahendra, Rooke, Heather M, Sipp, Douglas, Studer, Lorenz, Sugarman, Jeremy, Takahashi, Masayo, Zimmerman, Mark, and Kimmelman, Jonathan

Subjects
Stem Cells, Humans, Informed Consent, Societies, Scientific, Clinical Trials as Topic, Stem Cell Research, Translational Medical Research, EMRO, clinical translation, clinical trials, communication, embryo research, guidelines, stem cells
Abstract

The International Society for Stem Cell Research (ISSCR) presents its 2016 Guidelines for Stem Cell Research and Clinical Translation (ISSCR, 2016). The 2016 guidelines reflect the revision and extension of two past sets of guidelines (ISSCR, 2006; ISSCR, 2008) to address new and emerging areas of stem cell discovery and application and evolving ethical, social, and policy challenges. These guidelines provide an integrated set of principles and best practices to drive progress in basic, translational, and clinical research. The guidelines demand rigor, oversight, and transparency in all aspects of practice, providing confidence to practitioners and public alike that stem cell science can proceed efficiently and remain responsive to public and patient interests. Here, we highlight key elements and recommendations in the guidelines and summarize the recommendations and deliberations behind them.

Published
2016

24. Statistically based splicing detection reveals neural enrichment and tissue-specific induction of circular RNA during human fetal development.

Author

Szabo, Linda, Morey, Robert, Palpant, Nathan J, Wang, Peter L, Afari, Nastaran, Jiang, Chuan, Parast, Mana M, Murry, Charles E, Laurent, Louise C, and Salzman, Julia

Subjects
Fetal Heart, Brain, Hela Cells, Myocytes, Cardiac, Humans, Sodium-Calcium Exchanger, RNA, RNA Splice Sites, RNA, Small Nuclear, Models, Statistical, Genomics, Evolution, Molecular, Organ Specificity, RNA Splicing, Fetal Development, Algorithms, RNA, Circular, HeLa Cells, Genetics, 1.1 Normal biological development and functioning, Bioinformatics, Environmental Sciences, Biological Sciences, Information and Computing Sciences
Abstract

BackgroundThe pervasive expression of circular RNA is a recently discovered feature of gene expression in highly diverged eukaryotes, but the functions of most circular RNAs are still unknown. Computational methods to discover and quantify circular RNA are essential. Moreover, discovering biological contexts where circular RNAs are regulated will shed light on potential functional roles they may play.ResultsWe present a new algorithm that increases the sensitivity and specificity of circular RNA detection by discovering and quantifying circular and linear RNA splicing events at both annotated and un-annotated exon boundaries, including intergenic regions of the genome, with high statistical confidence. Unlike approaches that rely on read count and exon homology to determine confidence in prediction of circular RNA expression, our algorithm uses a statistical approach. Using our algorithm, we unveiled striking induction of general and tissue-specific circular RNAs, including in the heart and lung, during human fetal development. We discover regions of the human fetal brain, such as the frontal cortex, with marked enrichment for genes where circular RNA isoforms are dominant.ConclusionsThe vast majority of circular RNA production occurs at major spliceosome splice sites; however, we find the first examples of developmentally induced circular RNAs processed by the minor spliceosome, and an enriched propensity of minor spliceosome donors to splice into circular RNA at un-annotated, rather than annotated, exons. Together, these results suggest a potentially significant role for circular RNA in human development.

Published
2015

26. Quantifying Microvascular Structure in Healthy and Infarcted Rat Hearts Using Optical Coherence Tomography Angiography

Author

Xie, Zhiying, Zeinstra, Nicole, Kirby, Mitchell A., Le, Nhan Minh, Murry, Charles E., Zheng, Ying, and Wang, Ruikang K.

Abstract

Myocardial infarction (MI) is a life-threatening medical emergency resulting in coronary microvascular dysregulation and heart muscle damage. One of the primary characteristics of MI is capillary loss, which plays a significant role in the progression of this cardiovascular condition. In this study, we utilized optical coherence tomography angiography (OCTA) to image coronary microcirculation in fixed rat hearts, aiming to analyze coronary microvascular impairment post-infarction. Various angiographic metrics are presented to quantify vascular features, including the vessel area density, vessel complexity index, vessel tortuosity index, and flow impairment. Pathological differences identified from OCTA analysis are corroborated with histological analysis. The quantitative assessments reveal a significant decrease in microvascular density in the capillary-sized vessels and an enlargement for the arteriole/venule-sized vessels. Further, microvascular tortuosity and complexity exhibit an increase after myocardial infarction. The results underscore the feasibility of using OCTA to offer qualitative microvascular details and quantitative metrics, providing insights into coronary vascular network remodeling during disease progression and response to therapy.

Published
2024
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27. Dystrophin-deficient cardiomyocytes derived from human urine: New biologic reagents for drug discovery

Author

Guan, Xuan, Mack, David L, Moreno, Claudia M, Strande, Jennifer L, Mathieu, Julie, Shi, Yingai, Markert, Chad D, Wang, Zejing, Liu, Guihua, Lawlor, Michael W, Moorefield, Emily C, Jones, Tara N, Fugate, James A, Furth, Mark E, Murry, Charles E, Ruohola-Baker, Hannele, Zhang, Yuanyuan, Santana, Luis F, and Childers, Martin K

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Oncology and Carcinogenesis, Rare Diseases, Stem Cell Research - Induced Pluripotent Stem Cell - Non-Human, Cardiovascular, Stem Cell Research, Muscular Dystrophy, Pediatric, Intellectual and Developmental Disabilities (IDD), Regenerative Medicine, Stem Cell Research - Induced Pluripotent Stem Cell, Brain Disorders, Duchenne/ Becker Muscular Dystrophy, Stem Cell Research - Induced Pluripotent Stem Cell - Human, Adult, Animals, Case-Control Studies, Cell Differentiation, Cells, Cultured, Drug Discovery, Dystrophin, Female, Humans, Induced Pluripotent Stem Cells, Kruppel-Like Factor 4, Kruppel-Like Transcription Factors, Male, Mice, Mice, Inbred NOD, Mice, SCID, Muscular Dystrophy, Duchenne, Myocytes, Cardiac, Proto-Oncogene Proteins c-myc, Telomerase, Young Adult, Biological Sciences, Medical and Health Sciences, Developmental Biology, Genetics, Medical biotechnology, Oncology and carcinogenesis
Abstract

The ability to extract somatic cells from a patient and reprogram them to pluripotency opens up new possibilities for personalized medicine. Induced pluripotent stem cells (iPSCs) have been employed to generate beating cardiomyocytes from a patient's skin or blood cells. Here, iPSC methods were used to generate cardiomyocytes starting from the urine of a patient with Duchenne muscular dystrophy (DMD). Urine was chosen as a starting material because it contains adult stem cells called urine-derived stem cells (USCs). USCs express the canonical reprogramming factors c-myc and klf4, and possess high telomerase activity. Pluripotency of urine-derived iPSC clones was confirmed by immunocytochemistry, RT-PCR and teratoma formation. Urine-derived iPSC clones generated from healthy volunteers and a DMD patient were differentiated into beating cardiomyocytes using a series of small molecules in monolayer culture. Results indicate that cardiomyocytes retain the DMD patient's dystrophin mutation. Physiological assays suggest that dystrophin-deficient cardiomyocytes possess phenotypic differences from normal cardiomyocytes. These results demonstrate the feasibility of generating cardiomyocytes from a urine sample and that urine-derived cardiomyocytes retain characteristic features that might be further exploited for mechanistic studies and drug discovery.

Published
2014

29. Human Organ-Specific Endothelial Cell Heterogeneity

Author

Marcu, Raluca, Choi, Yoon Jung, Xue, Jun, Fortin, Chelsea L., Wang, Yuliang, Nagao, Ryan J., Xu, Jin, MacDonald, James W., Bammler, Theo K., Murry, Charles E., Muczynski, Kimberly, Stevens, Kelly R., Himmelfarb, Jonathan, Schwartz, Stephen M., and Zheng, Ying

Published
2018
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33. Epicardial cells derived from human embryonic stem cells augment cardiomyocyte-driven heart regeneration

Author

Bargehr, Johannes, Ong, Lay Ping, Colzani, Maria, Davaapil, Hongorzul, Hofsteen, Peter, Bhandari, Shiv, Gambardella, Laure, Le Novère, Nicolas, Iyer, Dharini, Sampaziotis, Fotios, Weinberger, Florian, Bertero, Alessandro, Leonard, Andrea, Bernard, William G., Martinson, Amy, Figg, Nichola, Regnier, Michael, Bennett, Martin R., Murry, Charles E., and Sinha, Sanjay

Published
2019
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36. Human embryonic stem cell–derived cardiomyocytes restore function in infarcted hearts of non-human primates

Author

Liu, Yen-Wen, Chen, Billy, Yang, Xiulan, Fugate, James A, Kalucki, Faith A, Futakuchi-Tsuchida, Akiko, Couture, Larry, Vogel, Keith W, Astley, Clifford A, Baldessari, Audrey, Ogle, Jason, Don, Creighton W, Steinberg, Zachary L, Seslar, Stephen P, Tuck, Stephanie A, Tsuchida, Hiroshi, Naumova, Anna V, Dupras, Sarah K, Lyu, Milly S, Lee, James, Hailey, Dale W, Reinecke, Hans, Pabon, Lil, Fryer, Benjamin H, MacLellan, W Robb, Thies, R Scott, and Murry, Charles E

Published
2018
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37. Gene editing to prevent ventricular arrhythmias associated with cardiomyocyte cell therapy

Author

Marchiano, Silvia, primary, Nakamura, Kenta, additional, Reinecke, Hans, additional, Neidig, Lauren, additional, Lai, Michael, additional, Kadota, Shin, additional, Perbellini, Filippo, additional, Yang, Xiulan, additional, Klaiman, Jordan M., additional, Blakely, Leslie P., additional, Karbassi, Elaheh, additional, Fields, Paul A., additional, Fenix, Aidan M., additional, Beussman, Kevin M., additional, Jayabalu, Anu, additional, Kalucki, Faith A., additional, Potter, Jennifer C., additional, Futakuchi-Tsuchida, Akiko, additional, Weber, Gerhard J., additional, Dupras, Sarah, additional, Tsuchida, Hiroshi, additional, Pabon, Lil, additional, Wang, Lili, additional, Knollmann, Björn C., additional, Kattman, Steven, additional, Thies, R. Scott, additional, Sniadecki, Nathan, additional, MacLellan, W. Robb, additional, Bertero, Alessandro, additional, and Murry, Charles E., additional

Published
2023
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38. Cell based dATP delivery as a therapy for chronic heart failure.

Author

Mhatre, Ketaki N, primary, Mathieu, Julie, additional, Martinson, Amy, additional, Flint, Galina, additional, Blakley, Leslie P, additional, Tabesh, Arash, additional, Reinecke, Hans, additional, Yang, Xiulan, additional, Guan, Xuan, additional, Murali, Eesha, additional, Klaiman, Jordan M, additional, Odom, Guy L, additional, Brown, Mary Beth, additional, Tian, Rong, additional, Hauschka, Stephen D, additional, Raftery, Daniel, additional, Moussavi-Harami, Farid, additional, Regnier, Michael, additional, and Murry, Charles E, additional

Published
2023
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41. Confronting stem cell hype

Author

Caulfield, Timothy, Sipp, Douglas, Murry, Charles E., Daley, George Q., and Kimmelman, Jonathan

Published
2016

48. STEROID-SPARING IMMUNOSUPPRESSION FOR LONG TERM ALLOGENEIC STEM-CELL DERIVED CARDIOMYOCYTE TRANSPLANTATION IN MACAQUES

Author

Nakamura, Kenta, primary, Nakamura, Daisy, additional, Neidig, Lauren, additional, Tsuchida, Hiroshi, additional, Hong, Sogun, additional, Lin, Yongshun, additional, Klaiman, Jordan, additional, Kattman, Steven, additional, Thies, R. Scott, additional, Dunbar, Cynthia, additional, Murry, Charles E., additional, and MacLellan, William Robb, additional

Published
2023
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50. dATP elevation induces myocardial metabolic remodeling to support improved cardiac function

Author

Mhatre, Ketaki N., primary, Murray, Jason D., additional, Flint, Galina, additional, McMillen, Timothy S., additional, Weber, Gerhard, additional, Shakeri, Majid, additional, Tu, An-Yue, additional, Steczina, Sonette, additional, Weiss, Robert, additional, Marcinek, David J., additional, Murry, Charles E., additional, Raftery, Daniel, additional, Tian, Rong, additional, Moussavi-Harami, Farid, additional, and Regnier, Michael, additional

Published
2023
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