Scarless genome editing of human pluripotent stem cells via transient puromycin selection B Steyer, Q Bu, E Cory, K Jiang, S Duong, D Sinha, S Steltzer, D Gamm, ... Stem cell reports 10 (2), 642-654, 2018 | 73 | 2018 |
Human iPSC modeling reveals mutation-specific responses to gene therapy in a genotypically diverse dominant maculopathy D Sinha, B Steyer, PK Shahi, KP Mueller, R Valiauga, KL Edwards, ... The American Journal of Human Genetics 107 (2), 278-292, 2020 | 39 | 2020 |
Deep learning detects cardiotoxicity in a high-content screen with induced pluripotent stem cell-derived cardiomyocytes F Grafton, J Ho, S Ranjbarvaziri, F Farshidfar, A Budan, S Steltzer, ... Elife 10, e68714, 2021 | 29 | 2021 |
Reversal of cardiac hypertrophy with an optimized MYBPC3 gene therapy L Lombardi, A Greer-Short, EC Leon, TN Qureshi, A Greenwood, CA Reid, ... MOLECULAR THERAPY 29 (4), 256-256, 2021 | 3 | 2021 |
Deep Learning Predicts Patterns of Cardiotoxicity in a High-Content Screen Using Induced Pluripotent Stem Cell–Derived Cardiomyocytes F Grafton, J Ho, S Ranjbarvaziri, F Farshidfar, A Budan, S Steltzer, ... BioRxiv, 2021.03. 23.436666, 2021 | 2 | 2021 |
Human iPSC modeling reveals mutation-specific responses to gene therapy in Best disease D Sinha, B Steyer, PK Shahi, K Mueller, R Valiauga, KL Edwards, C Bacig, ... bioRxiv, 796581, 2019 | 2 | 2019 |
Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model ofMYBPC3-Associated Cardiomyopathy A Greer-Short, A Greenwood, EC Leon, TN Qureshi, K von Kraut, J Wong, ... | 1 | 2024 |
AAV9:PKP2 improves heart function and survival in a Pkp2-deficient mouse model of arrhythmogenic right ventricular cardiomyopathy I Wu, A Zeng, A Greer-Short, JA Aycinena, AE Tefera, R Shenwai, ... Communications Medicine 4 (1), 38, 2024 | | 2024 |
Gene Therapy Mediates Therapeutic Improvement in Cardiac Hypertrophy and Survival in a Murine Model of MYBPC3-Associated Cardiomyopathy LM Lombardi, A Greer-Short, A Greenwood, EC Leon, TN Qureshi, ... bioRxiv, 2024.02. 19.581102, 2024 | | 2024 |
Cardiac AAV9: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Remodeling, and Reduces Mortality in a Mouse Model of ARVC I Wu, A Greer-Short, A Aycinena, A Tefera, R Shenwai, F Farshidfar, ... | | 2023 |
Modeling Genetic Dilated Cardiomyopathies With Engineered Heart Tissues From Patient-derived And Isogenic Mutant Induced Pluripotent Stem Cells JH Tsui, B Zepeda, SS Steltzer, JJ Ho, J Priest, T Hoey CIRCULATION RESEARCH 131, 2022 | | 2022 |
Abstract P2121: Modeling Genetic Dilated Cardiomyopathies With Engineered Heart Tissues From Patient-derived And Isogenic Mutant Induced Pluripotent Stem Cells JH Tsui, B Zepeda, SS Steltzer, JJ Ho, J Priest, T Hoey Circulation Research 131 (Suppl_1), AP2121-AP2121, 2022 | | 2022 |
Cardiac AAV: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Right Ventricular Remodeling, Improves Heart Function, and Extends Survival in a Pkp2-deficient … I Wu, A Greer-Short, A Zeng, E Xu, M Van Pell, A Aycinena, N Getuiza, ... | | 2022 |
Cardiac AAV: PKP2 gene transfer prevents development of arrhythmogenic cardiomyopathy in a PKP2-deficient mouse model ZH Yang, J Yang, A Zeng, I Wu, A Greer-Short, A Aycinena, N Getuiza, ... HUMAN GENE THERAPY 32 (19-20), A43-A43, 2021 | | 2021 |
Prevention of Premature Lethality and Reversal of Cardiac Hypertrophy with an Optimized MYBPC3 Gene Therapy LM Lombardi, A Greer-Short, EC Leon, TN Qureshi, A Greenwood, ... HUMAN GENE THERAPY 32 (19-20), A10-A11, 2021 | | 2021 |
Modeling Genetic Dilated Cardiomyopathy Using Induced Pluripotent Stem Cell-derived Engineered Heart Tissues For Precision Medicine RE Butler, SS Steltzer, B Zepeda, JH Tsui, MA Mandegar, T Hoey, JJ Ho CIRCULATION RESEARCH 129, 2021 | | 2021 |
Abstract P436: Modeling Genetic Dilated Cardiomyopathy Using Induced Pluripotent Stem Cell-derived Engineered Heart Tissues For Precision Medicine RE Butler, SS Steltzer, B Zepeda, JH Tsui, MA Mandegar, T Hoey, JJ Ho Circulation Research 129 (Suppl_1), AP436-AP436, 2021 | | 2021 |