Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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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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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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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.
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Parajuli S, Gallagher T, Flotte TR. Immune Toxicities in AAV Gene Therapy: Overview for Clinicians. Int J Mol Sci. 2026 Mar 31; 27(7).
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Iyer S, Daman K, Sun Y, Tutto A, Holbrook SE, Joynt AT, Yan J, Ambegaokar P, Guo D, Liu P, Stauffer JE, Maitland SA, Lee SM, Xiao Y, Huang HC, Zhu LJ, Gallagher TL, Cox GA, Keeler AM, Siegwart DJ, Emerson CP, Wolfe SA. SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model. Mol Ther. 2026 Jul 01; 34(7):3885-3902.
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Saylam E, D'ambrosio ES, Pesco MT, Gushchina LV. Adeno-Associated Virus Toxicity in Duchenne Muscular Dystrophy: Mechanisms and Clinical Considerations. Genes (Basel). 2026 Feb 27; 17(3).
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Montano-Campos JF, Adair JE, Basu A, Kyeyune RB, Bayigga L, Kityo-Mutuluuza C, Hansen R. Cost-effectiveness of gene therapy for sickle cell disease in Uganda: tailoring high-income evidence to Uganda's context. Gene Ther. 2026 Mar; 33(2):109-117.
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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.