Alanine expansions of PABPN1 cause oculopharyngeal muscular dystrophy. Administration of a single AAV9 vector expressing a corrected version of the PABPN1 gene and two siRNAs in miRNA backbones to silence endogenous PABPN1 can restore muscle size, weight, and force in an OPMD surrogate mouse model to wild-type levels.
BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy
Cappellari O.;
2021-01-01
Abstract
Alanine expansions of PABPN1 cause oculopharyngeal muscular dystrophy. Administration of a single AAV9 vector expressing a corrected version of the PABPN1 gene and two siRNAs in miRNA backbones to silence endogenous PABPN1 can restore muscle size, weight, and force in an OPMD surrogate mouse model to wild-type levels.File in questo prodotto:
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