VARIANT RNAI AGAINST ALPHA-SYNUCLEIN

VARIANT RNAI AGAINST ALPHA-SYNUCLEIN

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 con...

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Hauptverfasser: RICHARDS, Brenda, LATTA-MAHIEU, Martine, BLANCHARD, Véronique, OBINU, Maria Carmen, ELMER, Bradford, TAUPIN, Véronique
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung: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.