PDE10A Inhibition Reduces the Manifestation of Pathology in DMD Zebrafish and Represses the Genetic Modifier PITPNA

Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene. Absence of dystrophin protein leads to progressive degradation of skeletal and cardiac function and leads to premature death. Over the years, zebrafish have been increasingly used for studying DMD and...

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Detalles Bibliográficos
Autores principales: Lambert, Matthias R., Spinazzola, Janelle M., Widrick, Jeffrey J., Pakula, Anna, Conner, James R., Chin, Janice E., Owens, Jane M., Kunkel, Louis M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7934586/
https://www.ncbi.nlm.nih.gov/pubmed/33221436
http://dx.doi.org/10.1016/j.ymthe.2020.11.021

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