Variant RNAi against alpha-synuclein
AssigneeGenzyme Corporation
InventorsBradford Elmer, Brenda Richards, Martine Latta-Mahieu, Maria Carmen Obinu, Véronique Taupin, Véronique Blanchard
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Abstract
Provided herein are RNAi molecules for treating neurodegenerative synucleinopathies. In some embodiments, the RNAi molecules target expression of alpha-synuclein (SNCA). Further provided herein are expression constructs, vectors (e.g. rAAV), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat neurodegenerative synucleinopathies including Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies.
Claims12
An RNAi comprising a first strand and a second strand, wherein:
- a) the first strand and the second strand form a duplex;
- b) the first strand comprises a guide region, wherein the guide region comprises a nucleic acid having the sequence 5′-ACUGCGCACAUUGGAACUGAG-3′ (SEQ ID NO:
- 29);
- and
- c) the second strand comprises a non-guide region.
Dependencies are inferred from the claim text.