Gene therapy
AssigneeOxford University Innovation Limited
InventorsHarry ORLANS, Robert E. MACLAREN
Life of this patent
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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.
Claims15
A gene therapy vector that expresses a rhodopsin transgene;
- wherein said transgene encodes functional rhodopsin;
- wherein the coding region of said rhodopsin transgene does not comprise the sequences of SEQ ID NOs:
- 5 or 7;
- and wherein the 5′UTR of the rhodopsin transgene consists of a 5′ end consisting of the sequence of nucleotides 1 to 423 of SEQ ID NO:
- 39;
- a 3′ end consisting of the sequence of nucleotides 424 to 439 of SEQ ID NO:
- 39;
- and a sequence inserted between nucleotides 1 to 423 and 424 to 439 of SEQ ID NO:
- 39;
- wherein said inserted sequence consists of SEQ ID NO:
- 26.
A gene therapy vector that expresses a rhodopsin transgene;
- wherein said transgene encodes functional rhodopsin;
- wherein the coding region of said rhodopsin transgene does not comprise the sequences of SEQ ID NOs:
- 5 or 7;
- and wherein the 5′UTR of the rhodopsin transgene consists of a 5′ end consisting of the sequence of nucleotides 1 to 423 of SEQ ID NO:
- 39;
- a 3′ end consisting of the sequence of nucleotides 424 to 439 of SEQ ID NO:
- 39;
- and a sequence inserted between nucleotides 1 to 423 and 424 to 439 of SEQ ID NO:
- 39;
- wherein said inserted sequence consists of, in a 5′ to 3′ direction:
- (a) SEQ ID NO:
- 26, (b) ATC, (c) SEQ ID NO:
- 37, (d) SEQ ID NO 12, and (e) SEQ ID NO:
- 38.
Dependencies are inferred from the claim text.