Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice

Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart failure and sudden cardiac death. In this study, we assessed two different genetic therapies—an adenine base editor (ABE8e...

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Detalles Bibliográficos
Autores principales: Reichart, Daniel, Newby, Gregory A., Wakimoto, Hiroko, Lun, Mingyue, Gorham, Joshua M., Curran, Justin J., Raguram, Aditya, DeLaughter, Daniel M., Conner, David A., Marsiglia, Júlia D. C., Kohli, Sajeev, Chmatal, Lukas, Page, David C., Zabaleta, Nerea, Vandenberghe, Luk, Liu, David R., Seidman, Jonathan G., Seidman, Christine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9941048/
https://www.ncbi.nlm.nih.gov/pubmed/36797483
http://dx.doi.org/10.1038/s41591-022-02190-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9941048/
https://www.ncbi.nlm.nih.gov/pubmed/36797483
http://dx.doi.org/10.1038/s41591-022-02190-7

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