A recombinant adeno-associated virus (rAAV) comprising an AAVhu68 capsid and a vector genome comprising a lysosomal beta-galactosidase gene (for example, galactosidase beta 1 gene, GBL1) is provided (i.e., rAAVhu68.GBL1). Also provided a composition containing an effective amount of rAAVhu68.GBL1 to ameliorate symptoms of GM1 gangliosidosis, including, e.g., increased average life span, decreased need for feeding tube, reduction in seizure incidence and frequency, reduction in progression towards neurocognitive decline and/or improvement in neurocognitive development.

Disclosed are compositions and methods of treating a neurodegenerative disease in an individual. The methods disclose administration of an Integrin α4β1, Very Late Antigen-4 positive neural precursor cell (“VLA4+NPC”) transfected with a lentivirus overexpressing wild type GCase to an individual having a neurodegenerative disorder. The neurodegenerative disease may include lipid storage diseases, for example Gaucher disease, Parkinson's disease (PD), Dementia with Lewy bodies.

Provided herein are novel AAV capsids and rAAV comprising the same. In one embodiment, vectors employing the AAV capsid show increased transduction in a selected tissue as compared to a prior art AAV.

Regimens useful in reducing the frequency of apheresis in a human patient having familial hypercholesterolemia are described. The method involves administering to the human subject via a peripheral vein by infusion of a suspension of replication deficient recombinant adeno-associated virus (rAAV).

Disclosed herein are compositions and in vitro and in vivo methods for reprogramming post-natal (adult and juvenile) tissue into insulinogenic cells. These compositions and methods are useful for a variety of purposes, including the development of diabetes therapies.

A pharmaceutical composition formulated for delivery of a recombinant adeno-associated virus (rAAV) vector comprising an AAV capsid and a vector genome having human galactosylceramidase (GALC) coding sequence is provided. Also provided are 5 methods and uses of a pharmaceutical composition comprising a rAAV for the treatment of Krabbe disease.

Provided herein is a method for treating and/or preventing various diseases including a decrease in glucose tolerance and a decrease in cognitive ability associated with aging with adipocytes over-expressing FFAR4 and a transplant composition including the adipocytes.

Methods and compositions are provided for activating transcription in a mammalian cell.

The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.

This application relates to therapeutic siRNA agents and methods of making and using the agents.