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RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 C.607 G > A in Vitro.

Natalia V. Klementieva,Evgenii A. Lunev,Anna A. Shmidt, Elizaveta M. Loseva,Irina M. Savchenko, Ekaterina A. Svetlova,Ivan I. Galkin,Anna V. Polikarpova, Evgeny V. Usachev,Svetlana G. Vassilieva, Valeria I. Marina, Marina A. Dzhenkova, Anna D. Romanova, Anton V. Agutin,Anna A. Timakova,Denis A. Reshetov,Tatiana V. Egorova,Maryana V. Bardina

Nucleic Acid Therapeutics（2024）

引用 1|浏览34
关键词
GNAO1 encephalopathy,RNA therapeutics,gene suppression,RNA interference,vectorized RNAi,adeno-associated virus vectors
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