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• W2912631497 abstract "Abstract There is an urgent need to develop the next-generation vectors for gene therapy of muscle disorders, given the relatively modest advances in clinical trials. These vectors should express substantially higher levels of the therapeutic transgene, enabling the use of lower and safer vector doses. In the current study, we identify potent muscle-specific transcriptional cis -regulatory modules ( CRMs ), containing clusters of transcription factor binding sites, using a genome-wide data-mining strategy. These novel muscle-specific CRMs result in a substantial increase in muscle-specific gene transcription (up to 400-fold) when delivered using adeno-associated viral vectors in mice. Significantly higher and sustained human micro-dystrophin and follistatin expression levels are attained than when conventional promoters are used. This results in robust phenotypic correction in dystrophic mice, without triggering apoptosis or evoking an immune response. This multidisciplinary approach has potentially broad implications for augmenting the efficacy and safety of muscle-directed gene therapy." @default.
• W2912631497 created "2019-02-21" @default.
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• W2912631497 date "2019-01-30" @default.
• W2912631497 modified "2023-10-16" @default.
• W2912631497 title "Next-generation muscle-directed gene therapy by in silico vector design" @default.
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• W2912631497 doi "https://doi.org/10.1038/s41467-018-08283-7" @default.
• W2912631497 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/6353880" @default.
• W2912631497 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/30700722" @default.
• W2912631497 hasPublicationYear "2019" @default.
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