AAV-mediated exon skipping therapy for Usher syndrome, type 2A.
AAV介导的Usher综合征2A型外显子跳跃疗法。
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.09.038
Mauriac Stephanie A, Lee Jiyoon, Zhang Jingyuan, Jin Jiahe, Nist-Lund Carl, Martin Camille T, Von Muhlenbrock Cristobal R, O'Malley Sydney, Chaves Mary G, Madison Margot A, Foster Kate, Long Shinong, Jacobs Chris, Kumar Sripriya Ravindra, Ramirez Silvia, Ichida Justin, Koehler Karl R, Holt Jeffrey R, Géléoc Gwenaëlle S G