The present invention provides methods and compositions comprising an adeno-associated virus (AAV) capsid protein, comprising one or more amino acids substitutions, wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.

The present invention provides methods and compositions comprising an adeno-associated virus (AAV) capsid protein, comprising one or more amino acids substitutions, wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.

The present invention comprises a method to diminish and/or eliminate atherosclerotic plaques, in mammals, through direct and indirect treatment of these plaques, in situ, using suitable substances which are capable of lipid removal, primarily through hydrolysis, either by a catalytic or stoichiometric process, wherein the substance targets receptors in and/or on the cell which lead to uptake into the lysosome. Such substances used to diminish and/or eliminate atherosclerotic plaques are generally comprised of lipid hydrolyzing proteins and/or polypeptides.

The invention provides methods for enhancing the delivery of viral vectors to the eye of a subject by administering a proteasome inhibitor or and a viral vector ending a gene of interest to the eye.

The invention provides engineered RNA precursors that when expressed in a cell are processed by the cell to produce targeted small interfering RNAs (siRNAs) that selectively silence targeted genes (by cleaving specific mRNAs) using the cell's own RNA interference (RNAi) pathway. By introducing nucleic acid molecules that encode these engineered RNA precursors into cells in vivo with appropriate regulatory sequences, expression of the engineered RNA precursors can be selectively controlled both temporally and spatially, i.e., at particular times and/or in particular tissues, organs, or cells.

The invention provides compositions and methods for treating neuropathic pain. Specifically, the disclosure provides a polynucleotide comprising a trigeminal ganglion (TGG) or dorsal root ganglion (DRG) promoter operably linked to a recombinant nucleic acid encoding an endonuclease that binds to a nucleotide sequence in the human colony stimulating factor 1 (hCSF1) gene and a method of using the polynucleotide or a vector comprising the polynucleotide for treatment of neuropathic pain.

The invention provides compositions and methods of treating subjects afflicted with a photoreceptor disorder. Methods for treating a subject suffering from a disorder characterized by photoreceptor cell degeneration are provided, wherein a gene encoding a photosensitive protein is introduced into a retinal cell of a subject. In one aspect of the invention, the retinal cells which receive the photosensitive protein include non-photoreceptor cells such as horizontal cells, amacrine cells, bipolar cells, and ganglion cells.

The invention features methods for regulating vascular properties by controlling the membrane properties of endothelial cells using optogenetics and light. The invention features methods to transport therapeutics across the vascular barrier into tissues such as the brain and the lung, with high spatial and temporal precision, and for controlling vascular properties such as vascular tone, arterial diameter, and vascular growth.

The invention provides compositions and methods of treating subjects afflicted with a photoreceptor disorder. Methods for treating a subject suffering from a disorder characterized by photoreceptor cell degeneration are provided, wherein a gene encoding a photosensitive protein is introduced into a retinal cell of a subject. In one aspect of the invention, the retinal cells which receive the photosensitive protein include non-photoreceptor cells such as horizontal cells, amacrine cells, bipolar cells, and ganglion cells.

The present invention discloses the identification and isolation of novel MHC class II epitopes derived from the cancer antigen, NY ESO-1. The novel MHC class II epitopes from NY-EsO-1 are recognized by CD4.sup.+ T lymphocytes in an HLA class II restricted manner, in particular HLA-DR or HLA-DP restricted. The products of the gene are promising candidates for immunotherapeutic strategies for the prevention, treatment and diagnosis of patients with cancer.