Gene therapy with dysferlin dual vectors
AssigneeResearch Institute At Nationwide Children's Hospital
InventorLouise Rodino-Klapac
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Abstract
Recombinant polynucleotides encoding fragments of a human dysferlin protein are described herein. In addition, plasmids, viral vectors, dual vector systems, cells, and compositions comprising such recombinant polynucleotides are further described. Such recombinant polynucleotides, plasmids, viral vectors, dual vector systems, cells, and compositions may be used to treat dysferlinopathies.
Claims15
A dual adeno-associated viral (AAV) vector system comprising:
- (a) a first AAV vector, wherein the first AAV vector comprises a first recombinant polynucleotide comprising a fragment of a human dysferlin (hDYSF) gene, wherein the fragment consists of the nucleotide sequence of SEQ ID NO:
- 1;
- and
- (b) a second AAV vector, wherein the second AAV vector comprises a second recombinant polynucleotide comprising a fragment of a hDYSF gene, wherein the fragment consists of the nucleotide sequence of SEQ ID NO:
- 2.
An adeno-associated viral (AAV) packaging system comprising:
- (a) (i) a first plasmid comprising a first recombinant polynucleotide comprising a fragment of a human dysferlin (hDYSF) gene, wherein the fragment consists of the nucleotide sequence of SEQ ID NO:
- 1;
- and
- (ii) a second plasmid comprising a second recombinant polynucleotide comprising a fragment of a hDYSF gene, wherein the fragment consists of the nucleotide sequence of SEQ ID NO:
- 2;
- (b) an adenovirus helper plasmid;
- and
- (c) a rep-cap plasmid.
A method of treating a dysferlinopathy, comprising administering to a subject in need thereof:
- (a) an effective amount of a first recombinant polynucleotide, wherein the first recombinant polynucleotide comprises a fragment of a human dysferlin (hDYSF) gene, wherein the fragment consists of the nucleotide sequence of SEQ ID NO:
- 1;
- and
- (b) an effective amount of a second recombinant polynucleotide, wherein the second recombinant polynucleotide comprises a fragment of a hDYSF gene, wherein the fragment consists of the nucleotide sequence of SEQ ID NO:
- 2.
Dependencies are inferred from the claim text.