Disease exacerbation in MYOrganoids derived from Duchenne Muscular Dystrophy iPSC reveals limitations of microdystrophin therapeutic efficacy

Abstract excerpt

<title>Abstract</title> <p>Current gene therapy approaches for Duchenne muscular dystrophy (DMD) using AAV-mediated delivery of microdystrophin (µDys) have shown limited efficacy in patients, contrasting with the favorable outcomes observed in animal models. This discrepancy is partly due to the lack of models that replicate key pathogenic features associated with the severity of the human disease, such as fibros...

Topics

Open a Topic to create a Post that cites this publication.

• Disease Models, Animal
• Dystrophin
• Genetic Therapy
• Models, Biological
• Muscle Physiology and Disorders
• Muscular Dystrophies
• Mutation
• Utrophin

Identifiers and source

Literature Corpus work

fd0d5e2f-409a-5f6a-8368-5a2b405252bc

DOI

10.21203/rs.3.rs-4270736/v1