Back to results
US 12,529,051 B2Granted
Methods of treating amyotrophic lateral sclerosis (ALS)
AssigneeUniversity of Massachusetts
InventorsChristian Mueller, Abbas Abdallah
Life of this patent
Estimating the term…
Abstract
Aspects of the disclosure relate to recombinant gene editing complexes comprising a recombinant gene editing protein and guide RNA (gRNA) that specifically hybridizes to a region of a C90RF72 gene (e.g., a region flanking a G4C2 repeat or within a exonic region of the gene).
Claims10
A recombinant adeno-associated virus (rAAV) comprising:
- (i) an isolated nucleic acid comprising a transgene encoding a first guide RNA (gRNA) and a second gRNA that each specifically hybridize to a target nucleic acid sequence flanking opposite sides of a G4C2 repeat of a C9ORF72 gene, flanked by adeno-associated virus (AAV) inverted terminal repeats (ITRs);
- and
- (ii) at least one AAV capsid protein,
- wherein the first gRNA consists of SEQ ID NO:
- 2 and the second gRNA consists of SEQ ID NO:
- 3.
3 more elements
Dependencies are inferred from the claim text.