Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression

Tandem duplication mutations are increasingly found to be the direct cause of many rare heritable diseases, accounting for up to 10% of cases. Unfortunately, animal models recapitulating such mutations are scarce, limiting our ability to study them and develop genome editing therapies. Here, we desc...

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Detalles Bibliográficos
Autores principales: Maino, Eleonora, Wojtal, Daria, Evagelou, Sonia L, Farheen, Aiman, Wong, Tatianna W Y, Lindsay, Kyle, Scott, Ori, Rizvi, Samar Z, Hyatt, Elzbieta, Rok, Matthew, Visuvanathan, Shagana, Chiodo, Amanda, Schneeweiss, Michelle, Ivakine, Evgueni A, Cohn, Ronald D
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8103086/
https://www.ncbi.nlm.nih.gov/pubmed/33724658
http://dx.doi.org/10.15252/emmm.202013228

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8103086/
https://www.ncbi.nlm.nih.gov/pubmed/33724658
http://dx.doi.org/10.15252/emmm.202013228

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