Base editing-mediated splicing correction therapy for spinal muscular atrophy
Xiang Lin, Haizhu Chen, Ying‐Qian Lu, Shunyan Hong, Xinde Hu, Yanxia Gao, Lu‐Lu Lai, Jin-Jing Li, Zishuai Wang, Wenqin Ying, Lixiang Ma, Ning Wang, Erwei Zuo, Hui Yang, Wan‐Jin Chen
Topics & Concepts
Spinal muscular atrophyRNA splicingMedicineAtrophyPhysical medicine and rehabilitationNeurosciencePathologyPsychologyBiologyDiseaseGeneticsRNAGeneNeurogenetic and Muscular Disorders ResearchCRISPR and Genetic EngineeringRNA Research and Splicing