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Science Translational Medicine|July 26, 2019
AAV-delivered eCD4-Ig protects rhesus macaques from high-dose SIVmac239 challengesMatthew R Gardner, Christoph H Fellinger, Lisa M Kattenhorn, et al.Nature Cell Biology|February 5, 2020
LATS suppresses mTORC1 activity to directly coordinate Hippo and mTORC1 pathways in growth controlWenjian Gan, Xiaoming Dai, Xiangpeng Dai, et al.Molecular Therapy. Methods & Clinical Development|August 21, 2023
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulationManuela Corti, Barry J Byrne, Dominic J Gessler, et al.Nature Communications|November 12, 2022
Impaired mitochondrial oxidative metabolism in skeletal progenitor cells leads to musculoskeletal disintegrationChujiao Lin, Qiyuan Yang, Dongsheng Guo, et al.Nature Communications|November 2, 2021
Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivoRaed Ibraheim, Phillip W L Tai, Aamir Mir, et al.The Journal of Biological Chemistry|August 21, 2022
Paradoxical activation of transcription factor SREBP1c and de novo lipogenesis by hepatocyte-selective ATP-citrate lyase depletion in obese miceBatuhan Yenilmez, Mark Kelly, Guo-Fang Zhang, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|May 19, 2022
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disordersBeverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, et al.Cell|July 22, 2025
In vivo prime editing rescues alternating hemiplegia of childhood in miceAlexander A Sousa, Markus Terrey, Holt A Sakai, et al.Biorxiv : the Preprint Server for Biology|May 4, 2026
Durable protection against SIV challenge by adeno-associated virus delivery of Env-specific antibodiesNatasha M Clark, Nida K Keles, Grace N Hagedorn, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|October 9, 2015
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VIIBrittney L Gurda, Adrien De Guilhem De Lataillade, Peter Bell, et al.Pageof 33