Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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Tang JH, Taghian T, Jayakumar S, Parigi G, Luchinat C, Hall E, Gray-Edwards HL, Meade TJ. Bioresponsive MR Imaging Probes for Noninvasive Monitoring of AAV Gene Therapy. Bioconjug Chem. 2026 Mar 18; 37(3):545-552.
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Lewis CJ, D'Souza P, Johnston JM, Acosta MT, Farmer C, Baker EH, Crowell A, Mojica Y, Ashraf S, Joseph L, V?zina G, Quezado Z, Yousef MH, Vardar Z, Shazeeb MS, Corti M, Blackwood M, Coleman K, Batista R, Thurm A, De Boever E, Gahl WA, Byrne BJ, Flotte TR, Jiang X, Gross AL, Keeler AM, Gray-Edwards H, Martin DR, Sena-Esteves M, Tifft CJ. AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial. N Engl J Med. 2026 Mar 26; 394(12):1184-1194.
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Knoll RM, Myoung S, Kons ZA, Reinshagen KL, Kempfle JS. Radiologic Assessment of the Round Window Anatomy in Pediatric Patients Relevant to Gene Therapy and Inner Ear Drug Delivery. Otol Neurotol. 2025 Dec 01; 46(10):1289-1294.
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Barrett D, Cannon PM, Mingozzi F, Porteus M, Rivi?re I, Flotte TR. Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases. Mol Ther. 2025 Nov 05; 33(11):5316-5326.
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Lonfat N, Moreno-Leon L, Punzo C, Khanna H. Update on Gene Therapy Clinical Trials for Eye Diseases. Hum Gene Ther. 2025 Oct; 36(19-20):1287-1300.
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Xing R, Xu M, Reil D, Destefano A, Cui M, Liu N, Liang J, Xu G, Luo L, Xu M, Zhang F, Tai PWL, Wei Y, Gruntman AM, Flotte TR, Gao G, Wang D. A single amino acid variant in the variable region I of AAV capsid confers liver detargeting. PLoS Pathog. 2025 Sep; 21(9):e1013533.
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Yang YS, Kim MJ, Chaugule S, Mayer E, DeSouza N, Ma H, Xie J, Lee KY, Li S, Gravallese E, Gao G, Shim JH. Nature-inspired IL-1 targeted therapy to treat chronic inflammatory diseases. Mol Ther. 2025 Dec 03; 33(12):6379-6397.
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Bamidele N, Pires-Ferreira D, Zheng C, Destefano A, Cheng H, Tang Q, Cao Y, Xie J, Gao G, Gruntman A, Sontheimer E, Flotte TR, Xue W. A compact base editor rescues AATD-associated liver and lung disease in mouse models. Mol Ther. 2025 Nov 05; 33(11):5817-5828.
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Daci R, Shazeeb MS, Owusu-Adjei B, K?hn A, King R, Parzych J, Spanakis SG, Moser RP, Flotte TR, Cataltepe OI. Robot-assisted intrathalamic infusion for gene therapy in young children: surgical considerations. J Neurosurg Pediatr. 2025 Nov 01; 36(5):609-620.
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Eichler F, Cataltepe OI, Daci R, Puri AS, Taghian T, Jiang X, Shazeeb MS, Kuhn A, Hader A, Celik H, Vardar Z, Lewis CJ, Artinian R, Nagy A, Vachha B, Thompson R, Gallagher T, Bateman S, Parzych J, Spanakis SG, Vaughn TA, Pier K, De Boever E, Abbott MA, D Ambrosio E, Kokoski D, Blackwood M, Drummond E, Ratai EM, Townsend EL, McLaughlin H, Tifft CJ, Keeler AM, Sena-Esteves M, Gray-Edwards HL, Flotte TR. Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial. Nat Med. 2025 Sep; 31(9):2927-2935.