Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice

Aupy, P.; Zarrouki, F.; Sandro, Quentin; Gastaldi, Cécile; Buclez, Pierre-Olivier; Mamchaoui, K.; García, L.; Vaillend, C.; Goyenvalle, A.

doi:10.1016/j.omtm.2020.04.025

Public

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice.

Shared by NobleBlocks on May 4, 2020 • 12:00 AM UTC

Authors:

Aupy P

Zarrouki F

Sandro Q

Abstract

Gene therapy and antisense approaches hold promise for the treatment of Duchenne muscular dystrophy (DMD). The advantages of both therapeutic strategies can be combined by vectorizing antisense sequences into an adeno-associated virus (AAV) vector. We previously reported the efficacy of AAV-U7 small...

Subject

Gene therapy

Adeno-associated viral vector

Duchenne muscular dystrophy

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