Patent ID: 11969478
Assignee: UCL BUSINESS LTD
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A method of treating retinal dystrophy caused by Retinal pigment epithelium-specific 65 kDa protein (RPE65) deficiency in a patient in need thereof, the method comprising administering to the patient via direct retinal, subretinal, or intravitreal injection, a therapeutically effective amount of an AAV vector comprising an expression construct comprising a promoter and an operably linked polynucleotide sequence, wherein the promoter consists of:
(a) nucleotides 12-761 of SEQ ID NO:2, or
(b) SEQ ID NO:2;, wherein the operably linked polynucleotide sequence comprises the sequence of SEQ ID NO: 4; and wherein expression of the operably linked polynucleotide sequence results in treatment of retinal dystrophy in the patient.