The invention relates to the use of vectors to improve vision by restoring RPE phagocytosis of photoreceptor outer segments in a patient suffering from retinal dysfunction and/or degeneration.

Methods for treating bestrophinopathies are provided herein. The method includes, administering to an eye of the subject a dose of a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid sequence encoding a human BEST1 protein, wherein the subject has at least one mutant BEST1 allele. Also provided are methods for evaluating treatments for retinal degeneration.

Provided is an application of an amino acid as a vehicle for delivery of a nucleic acid into the nervous system. By local injection, preferably, combined with means of stereotactic injection, immunofluorescence staining, confocal microscopy, etc., the single-stranded oligonucleotides are efficiently delivered into brain cells, and the plasmid vectors are also delivered into astrocytes in mouse brain.

Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions are useful, for example, in gene therapy mediated ectopic expression of cell surface receptors and targeted delivery of compounds, such as conjugated oligonucleotides, to the non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions can be useful, for example, to treat, prevent, delay or ameliorate disease in an individual by targeted reduction of a gene of interest in the non-native cell ectopically expressing cell surface receptors.

The present disclosure provides constructs comprising a coding sequence operably linked to a promoter, wherein the coding sequence encodes a pendrin protein. Exemplary constructs include AAV constructs. Also provided are methods of using disclosed constructs for the treatment of hearing loss and/or deafness.

The present invention relates to compositions and methods for treating ocular disorders. More specifically, the method includes non-surgical administration of gene therapies to the suprachoroidal space of the eye of a subject who is suffering from an ocular disorder.

Compositions and methods are provided for reprogramming demal fibroblasts to exhibit neurogenic properties including increased cell expression of NGF and Nt3 in vivo. In accordance with one embodiment such compositions are used in conjunction with standard treatment for use in treating neuropathic pain and stabilizing or stimulating production of PGP9.5+ mature nerve fiber in a diabetic patient’s tissues.

The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle containing a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease.

This invention presents a sequence of the virus AAV/XBP1s-HA, method and its use in the improvement of cognitive functions, of memory and of learning, as presented in the in vivo studies in FIG. 12/17 right panel.

Compositions and methods are provided for Tat-inducible expression of a CRISPR-associated endonuclease by a truncated HIV LTR promoter containing at least a core region and a TAR region of a HIV LTR promoter. The compositions may be used as a therapeutic treatment for the treatment and/or prevention of HIV.