Below are the most recent publications written about "Genetic Vectors" 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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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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Hahn PA, Shandilya S, da Costa LAB, da Silva LCF, O'Hagan D, Liang B, Engelman K, Gardner MR, Gao G, Fuchs SP, Martinez-Navio JM, Desrosiers RC, Ardeshir A, Magnani DM, Martins MA. The Immunogenicity of AAV-Encoded HIV-1 bNAbs in Rhesus Macaques Is Unaffected by a Short Course of the Immunomodulator CTLA4Ig. AIDS Res Hum Retroviruses. 2025 Dec; 41(12):567-579.
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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.
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Ardeshir A, O'Hagan D, Mehta I, Shandilya S, Hopkins LLJ, Adamson L, Kuroda MJ, Hahn PA, da Costa LAB, Fuchs SP, Martinez-Navio JM, Gardner MR, Van Rompay KKA, Magnani DM, Lifson JD, Gao G, Farzan M, Desrosiers RC, Das J, Martins MA. Determinants of successful AAV-vectored delivery of HIV-1 bNAbs in early life. Nature. 2025 Sep; 645(8082):1020-1028.
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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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Wang Y, Liu J, Zhu S, Hu S, Chen X, Mandon E, Tran NT, Zhang S, Qi Y, Ma H, He R, Cao Y, Su Q, Gallagher TL, Li Z, Zhou C, Tai PWL, Gao G, Xie J. miR-375 protects against acetaminophen-induced acute liver failure by orchestrating pharmacogene expression. Mol Ther. 2025 Oct 01; 33(10):4874-4888.