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

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

Abstract 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...

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Bibliographic Details
Main Authors: Eleonora Maino, Daria Wojtal, Sonia L Evagelou, Aiman Farheen, Tatianna W Y Wong, Kyle Lindsay, Ori Scott, Samar Z Rizvi, Elzbieta Hyatt, Matthew Rok, Shagana Visuvanathan, Amanda Chiodo, Michelle Schneeweiss, Evgueni A Ivakine, Ronald D Cohn
Format: Article
Language:English
Published: Wiley 2021-05-01
Series:EMBO Molecular Medicine
Subjects:
AAVs
CRISPR/Cas9
Duchenne muscular dystrophy
duplication mutations
genome editing
Online Access:https://doi.org/10.15252/emmm.202013228

Internet

https://doi.org/10.15252/emmm.202013228

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