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US 12,668,617 B2Granted
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
Life of this patent
Estimating the term…
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.
Claims9
A polynucleotide comprising a first nucleotide sequence and a second nucleotide sequence, wherein the first nucleotide sequence comprises the nucleotide sequence of SEQ ID NO:
- 13 or SEQ ID NO:
- 14, and wherein the second nucleotide sequence comprises the nucleotide sequence of SEQ ID NO:
- 15 or SEQ ID NO:
- 16.
1 more element
A recombinant adeno-associated 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 SEQ ID NO:
- 25.
15 more elements
Dependencies are inferred from the claim text.