Article

Disease exacerbation by fibroblast inclusion in Duchenne Muscular Dystrophy MYOrganoids reveals limitations of microdystrophin therapeutic efficacy

2023-07-28

Abstract excerpt

<h4>SUMMARY</h4> 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 fibrosis and mus...

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Literature Corpus work: f78645c1-ad05-5f98-a9a4-48a81285df3c
DOI: 10.1101/2023.07.26.550063

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