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Investigative Ophthalmology & Visual Science|September 12, 2025
RP1 Dual-AAV Gene Therapy Preserves Retinal Structure and Ameliorates Photoreceptor Degeneration in a Murine Model of Retinitis PigmentosaFanfei Liu, Qiqi Li, Jing Su, et al.
Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics|September 19, 2025
Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfataseXiu Jin, Qin Ye, Xiaoyi Wu, et al.
Human Gene Therapy|March 26, 2026
Lipid Nanoparticle-Delivered mRNA Therapy Corrects Neonatal Murine MPS I-HJiamei Fu, Mei Luo, Xiu Jin, et al.
The New England Journal of Medicine|June 10, 2026
Subretinal Gene Therapy for X-Linked RetinoschisisLicong Liang, Kaiqin She, Chengda Ren, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 11, 2025
Comparative evaluation of liver-directed knockin strategies with viral and nonviral vectors in mouse inherited disease modelsXiu Jin, Xiaoyi Wu, Junwei Song, et al.
Nature Communications|December 17, 2025
AAV-delivered engineered suppressor tRNA rescues visual function in mice with an inherited retinal diseaseChengda Ren, Li Song, Ming Hu, et al.
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