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US 12,644,122 B2Granted
Compositions and methods for treating amyotrophic lateral sclerosis
AssigneesChildren's Medical Center Corporation, Dana-Farber Cancer Institute, Inc.
InventorsAlessandra Biffi, Marco Peviani, Francisco J. Molina-Estevez
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Abstract
The present disclosure features methods and compositions for treating amyotrophic lateral sclerosis (ALS). The disclosed methods comprise administering to a subject having or suspected of having ALS a hematopoietic stem progenitor cell expressing at least one neuroprotective agent. The compositions disclosed comprise hematopoietic stem progenitor cells transduced to express a neuroprotective agent.
Claims8
A short hairpin RNA molecule (shRNA) that targets a Nox2 polynucleotide, the shRNA comprising the following sequence (SEQ ID NO:
- 1):
- ▾
- 5′ C CCUCCU A -
- CUGG GCA-GUGCC CGCUNNNNNNNNNNNNNNNNNNN-
- -
- GACC CGU CACGG GUGANNNNNNNNNNNNNNNNNNN-
- 3′UC ---AUU A C -
- CAU
- NNCUC G
- U
- NNGAG G
- AUG.
9 more elements
Dependencies are inferred from the claim text.