Abstract: The disclosure provides composition comprising a nucleic acid sequence comprising (a) a sequence encoding a vitelliform macular dystrophy-2 (VMD2) promoter, and (b) a sequence encoding a Bestrophin-1 (BEST1) protein as well as the use of these compositions for the treatment of macular dystrophy in a subject comprising administration of the composition to an eye of a subject via a subretinal or a suprachoroidal route.

Abstract: The disclosure relates to compositions and methods for the treatment of Retinitis Pigmentosa through the administration of a rAAV vector comprising an RPGRORF15 sequence

Abstract: The present disclosure provides an adeno-associated viral (AAV) vector system for expressing a human ABCA4 protein in a target cell, the AAV vector system comprising a first AAV vector comprising a first nucleic acid sequence and a second AAV vector comprising a second nucleic acid sequence; wherein the first nucleic acid sequence comprises a 5? end portion of an ABCA4 coding sequence (CDS) and the second nucleic acid sequence comprises a 3? end portion of an ABCA4 CDS, and the 5? end portion and the 3? end portion together encompass the entire ABCA4 CDS; wherein the first nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 105 to 3597 of SEQ ID NO: 1; wherein the second nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 3806 to 6926 of SEQ ID NO: 1; wherein the first nucleic acid sequence and the second nucleic acid sequence each comprise a region of sequence overlap with the other; and wherein the region of sequence

Abstract: A method for determining the activity of Rab escort protein 1 (REP1) comprising the steps: (a) providing a sample comprising REP1; (b) contacting the sample of step (a) with Rab6a, Rab geranylgeranyltransferase (Rab GGTase) and a lipid donor substrate; and (c) detecting the lipidated Rab6a product.

Abstract: The disclosure provides composition comprising a nucleic acid sequence comprising (a) a sequence encoding a vitelliform macular dystrophy-2 (VMD2) promoter, and (b) a sequence encoding a Bestrophin-1 (BEST1) protein as well as the use of these compositions for the treatment of macular dystrophy in a subject comprising administration of the composition to an eye of a subject via a subretinal or a suprachoroidal route.

Abstract: The invention provides methods and compositions for detecting and/or quantifying modified nucleic acid oligonucleotides. These methods and compositions are useful for detecting and quantifying diagnostic and/or therapeutic synthetic modified oligonucleotides, such as aptamers, RNAi, siRNA, antisense oligonucleotides or ribozymes in a biological sample.

Abstract: Disclosed are oligonucleotides complementary to VEGF-specific nucleic acid useful in reducing the expression of VEGF. Also disclosed are pharmaceutical formulations containing such oligonucleotides useful for treating various disorders associated with neovascularization and angiogenesis.

Abstract: Vascular Endothelial Growth Factor (VEGF), also known as vascular permeability factor (VPF), has been shown to play in integral role in abnormal angiogenesis associated with a variety of pathological states. This disclosure presents compounds, compositions, and methods for inhibiting such abnormal angiogenesis. In particular, this disclosure presents several antisense oligonucleotides from 19 to 21 bases long that bind to VEGF RNA and inhibit production of the expression product. These antisense oligonucleotides are useful in the treatment of pathological states in which VEGF expression plays a role.

Abstract: Disclosed are oligonucleotides complementary to VEGF-specific nucleic acid useful in reducing the expression of VEGF. Also disclosed are pharmaceutical formulations containing such oligonucleotides useful for treating various disorders associated with neovascularization and angiogenesis, and methods for treating psoriasis.

Abstract: Disclosed are oligonucleotides complementary to VEGF-specific nucleic acid useful in reducing the expression of VEGF. Also disclosed are pharmaceutical formulations containing such oligonucleotides useful for treating various disorders associated with neovascularization and angiogenesis, and methods for treating psoriasis.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.

Abstract: Disclosed are oligonucleotides complementary to VEGF-specific nucleic acid useful in reducing the expression of VEGF. Also disclosed are pharmaceutical formulations containing such oligonucleotides useful for treating various disorders associated with neovascularization and angiogenesis.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.

Abstract: Disclosed are methods of reducing neovascularization and of treating various disorders associated with neovascularization. These methods include administering to a tissue or subject a synthetic oligonucleotide specific for vascular endothelial growth factor nucleic acid effective in inhibiting the expression of vascular endothelial growth factor.