CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability
CRISPR介导的转录激活作为一种不依赖于突变的SYNGAP1相关智力障碍治疗策略
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影响因子:
doi:10.1101/2025.10.28.685100
Sichlinger, Laura; Reilly, Molly B; Arora, Sakshi; Zhang, Shuo; Marotta, Nicolas; Rodríguez-Acevedo, Kiara L; Hooks, Marisol; Czarnecki, Kyle S; Winter, Julia J; Waxman, Elisa A; Dungan, Lea V; Hong, Ingie; Araki, Yoichi; Johnson, Richard; Huganir, Richard L; Pavani, Giulia; French, Deborah L; Davidson, Beverly L; Prosser, Benjamin L; Heller, Elizabeth A