Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model

Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model

Duchenne muscular dystrophy (DMD) is caused by mutations in the <i>DMD</i> gene that disrupt the open reading frame and thus prevent production of functional dystrophin proteins. Recent advances in DMD treatment, notably exon skipping and AAV gene therapy, have achieved some success aime...

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Bibliographic Details
Main Authors: Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P. Kelly, Federica Montanaro, Jennifer E. Morgan, Maaike van Putten, Annemieke Aartsma-Rus, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
Format: Article
Language:English
Published: MDPI AG 2023-12-01
Series:Biomedicines
Subjects:
antisense oligonucleotides
exon skipping
Duchenne muscular dystrophy
brain comorbidities
exon 53
CNS delivery
Online Access:https://www.mdpi.com/2227-9059/11/12/3243

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