AAV-BASED GENE THERAPY APPROACH FOR THE TREATMENT OF LGMD2B / DYSFERLINOPATHY

Abstract

The present invention relates to a composition comprising: a) a first nucleic acid molecule comprising: a sequence encoding a first portion of the dysferlin protein; and a first portion of a cleaved intein; and b) a second nucleic acid molecule comprising: a sequence encoding a second portion of the dysferlin protein; and a second portion of a cleaved intein; wherein the first nucleic acid molecule and the second nucleic acid molecule encode different portions of the dysferlin protein. The first and second portions of the dysferlin protein together provide a functional dysferlin protein. Furthermore, the first nucleic acid molecule is packaged into a first vector and the second nucleic acid molecule is packaged into a second vector, wherein each vector is an adeno-associated viral (AAV) vector. The composition is used to treat a subject suffering from dysferlinopathy.