Gene therapy
AssigneesThe University of Manchester, Centre Hospitalier Universitaire Sainte-Justine
InventorsAlexey Pchejetsky, Brian Bigger, Claire O'Leary
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Abstract
The present invention relates to compositions and adeno associated viral vectors comprising an optimised HGSNAT nucleic acid sequence of SEQ ID No. 1 or a derivative sequence having at least 77% homology thereof. Uses of such compositions and vectors are also contemplated along with kits of parts for their administration.
Claims31
A composition comprising an optimised HGSNAT nucleic acid sequence of SEQ ID No. 1 or a derivative sequence having at least 77% homology thereof.
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An adeno associated viral (AAV) vector comprising an optimised HGSNAT nucleic acid sequence of SEQ ID No. 1 or a derivative sequence having at least 77% homology thereof.
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Use of an adeno associated viral (AAV) vector having a AAV9, AAV8, AAV2 True Type or RH10 serotype for intracranial delivery of therapeutic nucleic acid sequence for the treatment of a disease or condition affecting the brain of an individual.
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Use of an adeno associated viral (AAV) vector having a AAV9 or AAV8 or AAV2 True Type or RH10 serotype for intracranial delivery of a HGSNAT cDNA nucleic acid sequence.
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Dependencies are inferred from the claim text.