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Article

Successful large gene augmentation of USH2A with non-viral episomal vectors.

Molecular therapy : the journal of the American Society of Gene Therapy - 6 Sept 2023

Toms Maria, Toualbi Lyes, Almeida Patrick V, Harbottle Richard, Moosajee Mariya

Abstract excerpt

USH2A mutations are a common cause of autosomal recessive retinitis pigmentosa (RP) and Usher syndrome, for which there are currently no approved treatments. Gene augmentation is a valuable therapeutic strategy for treating many inherited retinal diseases; however, conventional adeno-associated virus (AAV) gene therapy cannot accommodate cDNAs exceeding 4.7 kb, such as the 15.6-kb-long USH2A coding sequence. In...

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Identifiers

PMID: 37337429
DOI: 10.1016/j.ymthe.2023.06.012
PMCID: PMC10491995

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