Abstract: A method of treating a retinal disease in a subject in need thereof, the method comprising administering to the subject a vector that comprises a mirtron for knocking down expression of a target gene expressed in the retina and a gene therapy vector comprising a mirtron for rhodopsin knock-down.

Abstract: The invention provides methods for treating, preventing or reversing retinal degeneration. The methods administering to the subject a vector that expresses a Cadherin-related family member 1 (CDHR1) polypeptide. The invention also provides gene therapy vectors that expresses a CDHR1 polypeptide, host cells that express the gene therapy vectors, and pharmaceutical compositions comprising the gene therapy vector.

Abstract: The present invention relates to gene therapy for treatment or prevention of choroideremia.

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: 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: A method of treating a retinal disease in a subject in need thereof, the method comprising administering to the subject a vector that comprises a mirtron for knocking down expression of a target gene expressed in the retina and a gene therapy vector comprising a mirtron for rhodopsin knock-down.

Abstract: A method of gene therapy in a subject in need thereof, the method comprising administering to the subject a vector that comprises a transgene for expression in the subject and a mirtron for knocking down expression of a gene in the subject, wherein the mirtron is in the 5?UTR of the transgene. Also a vector comprising a transgene for expression from the vector and a mirtron for knocking down expression of a gene, wherein the mirtron is in the 5?UTR of the transgene and wherein the 5?UTR further comprises an exonic splice enhancer motif and/or wherein the mirtron is flanked in the 5?UTR by a fragment of the coding region of a reporter gene, and methods for preparing a vector.

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: A method of treating or preventing retinitis pigmentosa, wherein the method comprises administering an adeno-associated virus (AAV) vector comprising a nucleotide sequence encoding ciliary neurotrophic factor (CNTF) to a subject in need thereof.

Abstract: A method of treating an eye disease comprising administering an adeno-associated virus (AAV) vector to a mammalian subject by subretinal injection, wherein the AAV vector comprises a nucleotide sequence encoding melanopsin operably linked to an expression control sequence to promote expression of melanopsin in cells of the eye of the subject.