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Molecular Therapy : the Journal of the American Society of Gene Therapy|January 31, 2026
Modification of the VP1u region boosts transduction of adeno-associated virus vectors for ocular gene therapyMengtian Cui, Shun-Yun Cheng, Jialing Liang, et al.Elife|June 25, 2013
Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1Ling Luo, Hironori Uehara, Xiaohui Zhang, et al.Cell|February 5, 2025
Rhodoquinone carries electrons in the mammalian electron transport chainJonathan Valeros, Madison Jerome, Tenzin Tseyang, et al.The Journal of Clinical Investigation|September 30, 2025
Five-year analysis of efficacy and safety of a bidirectional AAV gene therapy in Tay-Sachs sheepToloo Taghian, Jillian Gallagher, Stephanie Bertrand, et al.Nature|February 25, 2015
AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challengesMatthew R Gardner, Lisa M Kattenhorn, Hema R Kondur, et al.Proceedings of the National Academy of Sciences of the United States of America|March 9, 2023
Divalent siRNAs are bioavailable in the lung and efficiently block SARS-CoV-2 infectionVignesh N Hariharan, Minwook Shin, Ching-Wen Chang, et al.Science Translational Medicine|February 11, 2021
Engineering adeno-associated viral vectors to evade innate immune and inflammatory responsesYing Kai Chan, Sean K Wang, Colin J Chu, et al.Nature|April 8, 2021
The NIH Somatic Cell Genome Editing programKrishanu Saha, Erik J Sontheimer, P J Brooks, et al.Pageof 33