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.
Claims15
A composition comprising an optimised heparin-α-glucosaminide N-acetyltransferase (HGSNAT) nucleic acid sequence of comprising the sequence SEQ ID No. 1 or a sequence having at least 77% homology thereof, wherein the sequence is optimised for enhanced expression or activity.
An adeno associated viral (AAV) vector comprising an optimised heparin-α-glucosaminide N-acetyltransferase (HGSNAT) nucleic acid sequence of comprising the sequence SEQ ID No. 1 or a sequence having at least 77% homology thereof, wherein the sequence is optimised for enhanced expression or activity.
Dependencies are inferred from the claim text.