RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
AssigneeUniversity of Massachusetts
InventorsChristian Mueller, Robert H. Brown, Jr.
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Abstract
The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.
Claims19
A synthetic miRNA that targets a C9orf72 RNA transcript, wherein the synthetic miRNA specifically binds to a nucleic acid sequence of the RNA transcript, wherein the nucleic acid sequence of the RNA transcript bound by the synthetic miRNA is encoded by a nucleic acid sequence set forth in SEQ ID NO:
- 2, 6, or 7, and wherein the synthetic miRNA further comprises flanking regions of miR-155.
A recombinant nucleic acid encoding a synthetic miRNA that targets a C9orf72 RNA transcript, wherein the synthetic miRNA specifically binds to a nucleic acid sequence of the RNA transcript, wherein the nucleic acid sequence of the RNA transcript bound by the synthetic miRNA is encoded by a nucleic acid sequence set forth in SEQ ID NO:
- 2, 6, or 7, and wherein the nucleic acid sequence encoding the synthetic miRNA is flanked by AAV inverted terminal repeats (ITRs).
A recombinant adeno-associated virus (rAAV) comprising a recombinant nucleic acid encoding a synthetic miRNA that targets a C9orf72 RNA transcript, wherein the synthetic miRNA specifically binds to a nucleic acid sequence of the RNA transcript, wherein the nucleic acid sequence of the RNA transcript bound by the synthetic miRNA is encoded by a nucleic acid sequence set forth in SEQ ID NO:
- 2, 6, or 7, and further comprising AAV inverted terminal repeats (ITRs).
Dependencies are inferred from the claim text.