Abstract: Some embodiments relate to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating muscular dystrophy resulting from the duplication of DMD exon 2.

Abstract: The invention provides compositions and methods for stimulating the expression of the human frataxin gene. The compositions described herein can be used, for instance, to produce genes and RNA equivalents optimized for expression in a particular cell type. The compositions and methods that can be used for treating Frederich ataxia. Using the compositions and methods of the disclosure, a patient (e.g., a mammalian patient, such as a human patient) having Frederich ataxia may be administered a plasmid (e.g., a viral vector) that contains a human frataxin gene (hFXN) or an RNA equivalent thereof.

Abstract: The present disclosure relates to recombinant adeno-associated virus (AAV) vector serotype, wherein the capsid protein of AAV serotypes is mutated at single or multiple sites. The disclosure further relates to an improved transduction efficiency of these mutant AAV serotypes. The AAV serotypes disclosed are AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10. The instant disclosure relates to nucleotide sequences, recombinant vector, methods and kit thereof.

Abstract: The present disclosure relates to recombinant adeno-associated virus (AAV) vector serotype, wherein the capsid protein of AAV serotypes is mutated at single or multiple sites. The disclosure further relates to an improved transduction efficiency of these mutant AAV serotypes. The AAV serotypes disclosed are AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10. The instant disclosure relates to nucleotide sequences, recombinant vector, methods and kit thereof.