Abstract: Disclosed are nucleic acid constructs comprising a nucleic acid sequence encoding a vitelliform macular dystrophy-2 (VMD2) promoter operably linked to a nucleic acid sequence encoding Rap1a. Disclosed are vectors comprising the nucleic acid constructs disclosed herein. Disclosed are compositions comprising the disclosed nucleic acid constructs or vectors. Also disclosed are methods of treating a subject having age-related macular degeneration comprising administering one or more of the disclosed nucleic acid constructs, vectors, or compositions to a subject in need thereof.

Abstract: Provided herein are compositions and methods for gene and etiology-nonspecific and circuit-specific treatment of diseases, utilizing vectors for delivery of light-sensitive proteins to diseased and normal cells and tissues of interest.

Abstract: Provided herein are compositions and methods for gene and etiology-nonspecific and circuit-specific treatment of diseases, utilizing vectors for delivery of light-sensitive proteins to diseased and normal cells and tissues of interest.

Abstract: Provided are methods for the identification of novel genes involved in a variety of cellular processes, including retinal degeneration, retinal disease, cancer, memory and learning, amylotropic lateral sclerosis, and methods for the identification of the function of a variety of genes and gene fragments of unknown function. The genes thus identified, as well as the compositions used in the identification methods, are also provided.

Abstract: A method for treating an ocular disorder characterized by the defect or absence of a normal gene in the ocular cells of a human or animal subject involves administering to the subject by subretinal injection an effective amount of a recombinant adeno-associated virus carrying a nucleic acid sequence encoding the normal gene under the control of a promoter sequence which expresses the product of the gene in the ocular cells. The ocular cells are preferably retinal pigment epithelial (RPE) cells, and the gene is preferably an RPE-specific gene, e.g., RPE65. The promoter is one that can express the gene product in the RPE cells. Compositions for subretinal administration are useful in this method.

Abstract: Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012?1×1013 vector genomes/ml.

Abstract: Provided are methods for the identification of novel genes involved in a variety of cellular processes, including retinal degeneration, retinal disease, cancer, memory and learning, amylotropic lateral sclerosis, and methods for the identification of the function of a variety of genes and gene fragments of unknown function. The genes thus identified, as well as the compositions used in the identification methods, are also provided.

Abstract: Disclosed are methods for the use of therapeutic polypeptide-encoding polynucleotides in the creation of transformed host cells and transgenic animals is disclosed. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions comprising polynucleotide sequences that express one or more mammalian PEDF or anti-angiogenesis polypeptides is described. In particular, the invention provides gene therapy methods for the prevention, long-term treatment and/or amelioration of symptoms of a variety of conditions and disorders in a mammalian eye, including, for example blindness, loss of vision, retinal degeneration, macular degeneration, and related disorders resulting from retinal or choroidal neovascularization in affected individuals.

Abstract: Disclosed are serotype-specific recombinant adeno-associated viral (rAAV) vectors, as well as viral particles and compositions comprising them, useful in the expression of neurotherapeutic agents (including neurotherapeutic peptides and polypeptides) in selected mammalian neural cells, as well as tissues and organ systems comprising them. In particular embodiments, rAAV serotype 1 and serotype 5 vectors are disclosed useful for the delivery of therapeutic agents to neural cells of affected mammals.

Abstract: In accordance with the present invention is provided a device and method for delivering DNA for the purpose of gene therapy to specific regions within and around the eye.

Abstract: Disclosed are synthetic and "humanized" versions of green fluorescent protein (GFP) genes adapted for high level expression in mammalian cells, especially those of human origin. Base substitutions are made in various codons in order to change the codon usage to one more appropriate for expression in mammalian cells. Recombinant vectors carrying such humanized genes are also disclosed. In addition, various methods for using the efficient expression of humanized GFP in mammalian cells and in animals are described.