Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
AssigneeResearch Institute At Nationwide Children's Hospital
InventorPaul Taylor Martin
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Abstract
Products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies are provided. In the methods, a protein including a linker domain, such as the heparin-binding domain of Heparin-Binding Epidermal Growth Factor-Like Growth Factor (HBEGF), is delivered to patients.
Claims40
A polynucleotide encoding a protein comprising:
- a) a first domain comprising the heparin-binding domain of Heparin-Binding Epidermal Growth Factor-Like Growth Factor (HBEGF), and a second domain comprising the G1-G5 domain of the human laminin alpha 2 (LAMA2) gene;
- b) a first domain comprising the heparin-binding domain of HBEGF and the EGF-like domain of HBEGF, and a second domain comprising the G1-G5 domain of the human LAMA2 gene;
- c) a first domain comprising the heparin-binding domain of HBEGF, and a second domain comprising the G3-G5 domain of the human LAMA2 gene;
- or
- d) a first domain comprising the heparin-binding domain of HBEGF and the EGF-like domain of HBEGF, and a second domain comprising the G3-G5 domain of the human LAMA2 gene.
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A recombinant adeno-associate virus (rAAV), wherein the genome of the rAAV comprises
- (a) the polynucleotide sequence of SEQ ID NO:
- 1,
- (b) the polynucleotide sequence of SEQ ID NO:
- 3,
- (c) the polynucleotide sequence of SEQ ID NO:
- 5,
- (d) the polynucleotide sequence of SEQ ID NO:
- 7,
- (e) nucleotides 3590 to 8215 of SEQ ID NO:
- 2,
- (f) nucleotides 3590 to 8341 of SEQ ID NO:
- 4,
- (g) nucleotides 3609 to 6929 of SEQ ID NO:
- 6,
- (h) nucleotides 3590 to 7036 of SEQ ID NO:
- 8 or
- (i) the nucleotide sequence set out in FIG. 13.
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A polynucleotide encoding a protein comprising:
- a) a first domain comprising the heparin-binding domain of HBEGF and a second domain comprising the processed native alpha chain of the human dystroglycan gene (DAG1alpha);
- or
- b) a first domain comprising the heparin-binding domain of HBEGF and the EGF-like domain of HBEGF, and a second domain comprising DAG1alpha.
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A recombinant adeno-associate virus (rAAV), wherein the genome of the rAAV comprises
- a) a polynucleotide sequence of SEQ ID NO:
- 9 or SEQ ID NO:
- 11,
- b) nucleotides 3590 to 6340 of SEQ ID NO:
- 10,
- c) nucleotides 3590 to 6049 of SEQ ID NO:
- 12 or
- d) the nucleotide sequence set out in FIG. 14.
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Dependencies are inferred from the claim text.