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US 12,612,647 B2Granted
Compositions useful in treatment of spinal muscular atrophy
AssigneeThe Trustees of the University of Pennsylvania
InventorsJames M. Wilson, Christian Hinderer, Nathan Katz, Qiang Wang
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Abstract
A rAAV vector is described herein which has an AAVhu68 capsid and at least one expression cassette in the capsid. The at least one expression cassette comprises nucleic acid sequences encoding a functional SMN protein and expression control sequences that direct expression of the SMN sequences in a host cell. Also provided are compositions containing this rAAVhu68.SMN vector and methods of using same for spinal muscular atrophy in a patient.
Claims8
A production cell in culture comprising:
- (a) an AAVhu68 VP1 nucleic acid sequence of SEQ ID NO:
- 7 or a sequence at least 99% identical thereto encoding a AAVhu68 VP1 protein of SEQ ID NO:
- 8, operably linked to a regulatory sequence which directs expression of AAVhu68 VP1, VP2 and VP3 proteins which assemble into an AAVhu68 capsid in the production cell;
- (b) a nucleic acid molecule comprising a vector genome for packaging into the AAVhu68 capsid comprising:
- an AAV 5′ inverted terminal repeat (ITR) sequence, a CB7 promoter comprising a human cytomegalovirus immediate early enhancer, a chicken beta actin promoter, an intron, a coding sequence for spinal motor neuron 1 (SMN1) isoform D which is SEQ ID NO:
- 1, a polyA, and an AAV 3′ ITR;
- and
- (c) AAV rep functions and helper functions necessary for packaging of the vector genome of (b) into the AAVhu68 capsid in the production cell to provide a recombinant AAV vector having the vector genome comprising the SMN1 coding sequences in the AAVhu68 capsid.
5 more elements
Dependencies are inferred from the claim text.