AAV1-U7

AAV1-U7 is a gene therapy product consisting of an adeno-associated virus serotype 1 (AAV1) vector engineered to express a modified small nuclear RNA (U7-snRNA) carrying antisense sequences designed to induce exon skipping in dystrophin pre-mRNA. The main mechanism is to promote the production of functional, albeit truncated, dystrophin protein in muscle fibers by redirecting the splicing machinery, specifically for the treatment of Duchenne muscular dystrophy (DMD). The therapy aims to restore the reading frame of the dystrophin transcript, enabling synthesis of a protein variant that partly compensates for the genetic defect. Preclinical studies in dystrophic mice and dogs have demonstrated that AAV1-U7 promotes significant dystrophin rescue and improvement of muscle function, though the longevity of expression is limited by loss of vector genomes from the muscle, particularly under dystrophic conditions[1][2][3][4][5].

Other names

Adeno-associated viral vector containing a modified U7-snRNA gene