Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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Wan F, He J, Ma H, PiresFerreira D, Kumanan V, Lee JS, Chen X, He R, Su Q, Gallagher TL, Zhu S, Cabrera GT, Zhao L, Shen J, Gruntman A, Brown RH, Xu Z, Gao G, Xie J. Intravenous administration of an engineered AAV9-gene-silencing vector suppresses human SOD1 and extends survival in an ALS mouse model. Nat Commun. 2026 06 25; 17(1).
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Fankhauser V, Kempfle JS. [Cell and gene therapies in Meni?re's disease: perspectives for a?slowly progressive inner ear disease]. HNO. 2026 Aug; 74(8):513-521.
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Chen X, Xie Q, Nath SJ, Tang M, Ma H, G?nes Y?, Sharma T, Liu H, Cui M, Du A, Lu M, Liu SY, Wassamon B, Xu M, Wu JY, Su Q, Fitzgibbons TP, Liu J, Wan F, Kumanan V, He R, Ma Y, Yang J, Gray-Edwards HL, Gallagher TL, Tai PWL, Gao G, Xie J. Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice. Nat Commun. 2026 May 23; 17(1).
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Nolen N, Aartsma-Rus A, Caneva C, Coughlin Ii CR, Cousin MA, Douthwright C, Graessner H, Kim-McManus O, Kuniholm A, Leonard S, Martinsen A, Meserve M, Synofzik M, Yandava B, Yu TW, Demarest S, Paxton RJ. Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies. Orphanet J Rare Dis. 2026 May 21; 21(1).
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Tang Q, Pires-Ferreira D, Mueller C, Gruntman AM, Flotte TR. Optimization of Alpha-1 Antitrypsin Expression from Adeno-Associated Virus Vectors. Hum Gene Ther. 2026 Jul; 37(13-14):609-617.
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Wang J, Turgeon CT, Loken PR, Gray-Edwards H, Gao G, Tortorelli S, Wang D, Strauss KA. Systemic dual-gene therapy reverses biochemical intoxication in the central metabolic compartment of Bckdha-/- mice. Mol Ther. 2026 Aug 05; 34(8):4569-4580.
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Brigatti KW, Rodrigues A, Sweigert E, Williamson J, Koehler A, Meier GL, Poskitt LE, Carson VJ, Robinson D, Strauss KA. Trial-ready external controls for gene therapy: The MATCH cohort in maple syrup urine disease. Cell Rep Med. 2026 Jun 16; 7(6):102799.
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Arjomandnejad M, Essien K, Sylvia K, Blackwood M, Tutto A, Katz E, Tang Q, Harris JE, Keeler AM. AAVrh32.33 capsid demonstrates unexpected dermal tropism regardless of immunodominant epitope. Mol Ther. 2026 Aug 05; 34(8):4531-4545.
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Wu HL, Webb GM, Fuchs SP, Pessoa CT, Hwang JM, Fisher HK, McCullen AJ, Humkey MC, Brown MK, Waytashek CM, Boyle CD, Kukula KA, Potter SK, Devlin G, Elmore ZC, Hull JA, Zikos J, Swanson T, Armantrout K, Chun K, Crank H, Bailey L, White R, Barber-Axthelm A, Fischer M, Smedley JV, Axthelm MK, Fennessey CM, Keele BF, Desrosiers RC, Magnani DM, Asokan A, Sacha JB. Adeno-associated virus gene therapy-mediated CCR5 blockade suppresses virus replication long term in SHIV-infected macaques. Sci Transl Med. 2026 Apr 29; 18(847):eadw1976.
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Xicluna R, Yamada K, Gonzalez-Visiedos M, Greenwood CM, Zaugg D, Waiz D, Steiner G, Hahn K, Schwandt T, Otteneder MB, Biswas M, Mu?oz-Melero M, Kumar SRP, Herzog RW, Haegel H. Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer. Mol Ther. 2026 Jul 01; 34(7):3805-3819.