RECOMBINANT ADENO-ASSOCIATED VIRUS DELIVERY OF EXON 2-TARGETED U7SNRNA POLYNUCLEOTIDE CONSTRUCTS
First Claim
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1. A method of ameliorating Duchenne Muscular Dystrophy in a patient with DMD exon 2 duplications in need thereof comprising the step of administering a recombinant adeno-associated virus (rAAV) to the patient, wherein the genome of the rAAV comprises at least one exon 2-targeted U7snRNA polynucleotide construct.
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Abstract
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
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Citations
18 Claims
- 1. A method of ameliorating Duchenne Muscular Dystrophy in a patient with DMD exon 2 duplications in need thereof comprising the step of administering a recombinant adeno-associated virus (rAAV) to the patient, wherein the genome of the rAAV comprises at least one exon 2-targeted U7snRNA polynucleotide construct.
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2. A method of inhibiting the progression of dystrophic pathology associated with Duchenne Muscular Dystrophy in a patient with DMD exon 2 duplications in need thereof comprising the step of administering a rAAV to the patient, wherein the genome of the rAAV comprises at least one exon 2-targeted U7snRNA polynucleotide construct.
- 3. A method of improving muscle function in a patient afflicted with Duchenne Muscular Dystrophy associated with DMD exon 2 duplications comprising the step of administering a rAAV to the patient, wherein the genome of the rAAV comprises at least one exon 2-targeted U7snRNA polynucleotide construct.
- 9. A method of delivering an exon 2-targeted U7snRNA polynucleotide construct to an patient with DMD exon 2 duplications, comprising the step of administering a rAAV to the patient, wherein the genome of the rAAV comprises at least one exon 2-targeted U7snRNA polynucleotide construct.
- 14. A recombinant adeno-associated virus (AAV) comprising a genome comprising at least one exon 2-targeted U7snRNA polynucleotide construct.
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15. A recombinant adeno-associated virus (AAV) comprising:
- an AAV rh.74 capsid, an AAV6 capsid or an AAV9 capsid; and
a genome comprising at least one exon 2-targeted U7snRNA polynucleotide construct.
- an AAV rh.74 capsid, an AAV6 capsid or an AAV9 capsid; and
Specification