C12N2750/14071

Methods of vaccine administration

This invention relates to a method of treating a dog for canine diseases comprising administering to the dog therapeutically effective amounts of a vaccine, wherein the vaccine comprises viral antigens, a bacterin, or both, and wherein the vaccine is administered subcutaneously or orally according to the schedules provided herein.

ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR TARGETED GENE THERAPY

Described herein are compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with tropisms showing increased specificity and efficiency of viral transduction in targeted cell-types, for e.g., the brain, and lung. The rAAV compositions described herein also have tropisms showing decreased specificity and decreased efficiency of viral transduction in an off-target cell type, for e.g., the liver. The rAAV compositions described herein encapsidate a transgene, such a therapeutic nucleic acid. Upon systemic delivery to a subject, the rAAV is capable of increased specificity and increased transduction of the transgene in a target cell-type, as compared to a parental or reference AAV.

Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy

Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV. The disclosed methods reduce fibrosis and improve muscle function by restoring B-sarcoglycan expression in muscle tissues. The disclosure further provides preclinical studies in animal models, and a clinical trial involving systemic delivery of the vector to patients, which shows significant improvements in muscle pathology, force production, and overall activity in treated subjects.

Clinical parameters by expression of factor VIII

Described herein are constructs used for liver-specific expression of a transgene.

ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND THE TREATMENT OF MUSCULAR DYSTROPHY

Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.