The invention provide 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.

The invention relates to the use of a recombinant porcine adeno-associated virus (AAV) vector comprising a peptide-modified porcine AAV serotype 1 (AAVpol) capsid in gene therapy of muscle and/or central nervous system (CNS) disorders, in particular neuromuscular diseases such as genetic neuromuscular diseases.

Compositions, methods, and applications that increase the efficiency of nucleic acid transfection are provided. In one aspect, a pharmaceutical composition may include at least about 0.5 mg/ml concentration of a nucleic acid condensed with a cationic lipopolymer suspended in an isotonic solution, where the cationic lipopolymer includes a cationic polymer backbone having cholesterol and polyethylene glycol covalently attached thereto, and wherein the molar ratio of cholesterol to cationic polymer backbone is within a range of from about 0.1 to about 10, and the molar ratio of polyethylene glycol to cationic polymer backbone is within a range of from about 0.1 to about 10. The composition further may include a filler excipient.

Arrestin-1 variants having reduced inhibitory effects on enolase-1 catalytic activity are described. The arrestin-1 variants can be used in the treatment of retinal degenerative diseases.

Disclosed herein are compositions and methods for modulatimi the production of a protein in a target cell, The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.

Provided herein are methods for improving memory or cognitive function in a subject by administering a composition to the brain of the subject, where the composition comprises: i) a compound that increases expression of MALAT-1 long non-coding RNA, ii) a MALAT-1 long-coding RNA nucleic acid sequence, or iii) at least one MALAT-1 derived piRNA nucleic acid sequence. Also provided herein are methods of screening candidate compounds for their ability to modulate the expression of MALAT-1 long non-coding RNA in brain cells. In certain embodiments, such identified modulators that increase expression are further administered to the brain of a lab animal to determine the impact of such modulators on learning and memory.

A chimeric molecule of one or more proteins or peptides fused, complexed or linked to one or more anionic molecules. Efficient in vitro and in vivo delivery is attained by encapsulating these molecules in cationic lipids or cationic liposomes. Methods of treatment include the intracellular delivery of these molecules to a specific therapeutic target.

The present disclosure provides compositions comprising an opsin polypeptide and an arrestin polypeptide and their use thereof. Exemplary embodiments provide a composition including an opsin polypeptide, or an opsin polypeptide and an arrestin polypeptide, wherein at least one of the opsin or arrestin polypeptide comprises at least one mutation that increases a temporal resolution of the opsin polypeptide’s response to light. The opsin polypeptide and the arrestin polypeptide can be operably linked or separate. Additionally, use of said compositions for restoring retinal photosensitivity or treating a retinal degenerative condition is also provided.

The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.