Provided herein are methods of treating phenylketonuria by normalizing levels of amino acids, neurotransmitters, and neurotransmitter metabolites in a subject having phenylketonuria.

The present invention encompasses treatments for neurologic disorders with recombinant vims vectors encoding G-protein coupled receptors. In particular, the invention is directed to the treatment of addiction disorders including but not limited to alcohol addiction and opiate addiction.

A rAAV vector is described herein which has an AAVhu68 capsid and at least one expression cassette in the capsid. The at least one expression cassette comprises nucleic acid sequences encoding a functional SMN protein and expression control sequences that direct expression of the SMN sequences in a host cell. Also provided are compositions containing this rAAVhu68.SMN vector and methods of using same for spinal muscular atrophy in a patient.

The present disclosure provides for improved recombinant AAV therapies for the treatment of X-linked retinoschisis (XLRS). These therapies are designed for administration to subjects, such as human subjects, including humans diagnosed with or suffering from XLRS.

Viral vectors comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.

Engineered CRISPR-Cas9 nucleases with altered and improved PAM specificities and their use in genomic engineering, epigenomic engineering, and genome targeting.

The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.

The present invention relates to stable and high-producing site-specific integration (SSI) host cells, e. g. Chinese hamster ovary (CHO)-derived host cells, methods to produce and to use them.

The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

A method of inhibiting myostatin signaling via a myostatin splice variant-derived protein is provided. The protein and an expression system thereof are applicable to therapy for diseases in which myostatin is involved.