Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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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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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.
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Iwanowicz A, Boudi A, Seeley C, Sapp E, Miller R, Liu S, Chase K, Shing K, Batista AR, Siena-Esteves M, Aronin N, DiFiglia M, Kegel-Gleason KB. Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease?Mice. Hum Gene Ther. 2025 Aug; 36(15-16):1083-1094.
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Manek R, Huang W, Huang Y, Guo L, Cornell CS, Shazeeb MS, Verbitsky A, Jackson R, Johnson J, Berthelette P, Yu D, Pfister EL, Bangari D, Ying X, Kumar D, Mueller C, Kyostio-Moore S. Blood phenylalanine lowering partially reverses white matter changes in a mouse model of phenylketonuria. Mol Ther. 2025 Sep 03; 33(9):4303-4319.
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Liu H, Liu N, Zhou C, Du A, Kapadia M, Tai PWL, Barton E, Gao G, Wang D. High-purity AAV vector production utilizing recombination-dependent minicircle formation and genetic coupling. EMBO Mol Med. 2025 Jun; 17(6):1475-1494.
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Byrne BJ, Flanigan KM, Matesanz SE, Finkel RS, Waldrop MA, D'Ambrosio ES, Johnson NE, Smith BK, B?nnemann C, Carrig S, Rossano JW, Greenberg B, Lalaguna L, Lara-Pezzi E, Subramony S, Corti M, Mercado-Rodriguez C, Leon-Astudillo C, Ahrens-Nicklas R, Bharucha-Goebel D, Gao G, Gessler DJ, Hwu WL, Chien YH, Lee NC, Boye SL, Boye SE, George LA. Current clinical applications of AAV-mediated gene therapy. Mol Ther. 2025 Jun 04; 33(6):2479-2516.
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Roger AL, El Haddad L, Huston ML, Kehoe S, Le D, Khan M, Scarrow E, Gonzalez T, Benkert A, Asokan A, ElMallah MK. GAA replacement improves respiratory muscle, neural, and alveolar pathology in the pompe mouse. Respir Physiol Neurobiol. 2025 07; 335:104433.
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Belgrad J, McConnell E, Leonard S, Nolen N, Lauffer MC, Watts JK, Yu T, Yan WX, Aartsma-Rus A. The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing. Nucleic Acids Res. 2025 Apr 22; 53(8).
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Keeler AM, Zhan W, Ram S, Fitzgerald KA, Gao G. The curious case of AAV immunology. Mol Ther. 2025 May 07; 33(5):1946-1965.