Below are the most recent publications written about "Transgenes" by people in Profiles.
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Walkey CJ, Snow KJ, Bulcha J, Cox AR, Martinez AE, Ljungberg MC, Lanza DG, De Giorgi M, Chuecos MA, Alves-Bezerra M, Suarez CF, Hartig SM, Hilsenbeck SG, Hsu CW, Saville E, Gaitan Y, Duryea J, Hannigan S, Dickinson ME, Mirochnitchenko O, Wang D, Lutz CM, Heaney JD, Gao G, Murray SA, Lagor WR. A comprehensive atlas of AAV tropism in the mouse. Mol Ther. 2025 Mar 05; 33(3):1282-1299.
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Xie Q, Chen X, Ma H, Zhu Y, Ma Y, Jalinous L, Cox GF, Weaver F, Yang J, Kennedy Z, Gruntman A, Du A, Su Q, He R, Tai PW, Gao G, Xie J. Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor. EMBO Mol Med. 2024 Apr; 16(4):945-965.
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Tai PWL. Integration of Gene Therapy Vectors: A Risk Factor for Tumorigenesis or Another Commensal Property of Adeno-Associated Viruses That Benefits Long-Term Transgene Expression? Hum Gene Ther. 2023 11; 34(21-22):1074-1076.
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Lek A, Wong B, Keeler A, Blackwood M, Ma K, Huang S, Sylvia K, Batista AR, Artinian R, Kokoski D, Parajuli S, Putra J, Carreon CK, Lidov H, Woodman K, Pajusalu S, Spinazzola JM, Gallagher T, LaRovere J, Balderson D, Black L, Sutton K, Horgan R, Lek M, Flotte T. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. N Engl J Med. 2023 Sep 28; 389(13):1203-1210.
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Ling Q, Herstine JA, Bradbury A, Gray SJ. AAV-based in vivo gene therapy for neurological disorders. Nat Rev Drug Discov. 2023 10; 22(10):789-806.
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Davis-Gardner ME, Weber JA, Xie J, Pekrun K, Alexander EA, Weisgrau KL, Furlott JR, Rakasz EG, Kay MA, Gao G, Farzan M, Gardner MR. A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors. Front Immunol. 2023; 14:1105617.
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McElroy A, Sena-Esteves M, Arjomandnejad M, Keeler AM, Gray-Edwards HL. Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System. Hum Gene Ther. 2022 09; 33(17-18):889-892.
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Muhuri M, Levy DI, Schulz M, McCarty D, Gao G. Durability of transgene expression after rAAV gene therapy. Mol Ther. 2022 04 06; 30(4):1364-1380.
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Paes Dias M, Tripathi V, van der Heijden I, Cong K, Manolika EM, Bhin J, Gogola E, Galanos P, Annunziato S, Lieftink C, And?jar-S?nchez M, Chakrabarty S, Smith GCM, van de Ven M, Beijersbergen RL, Bartkova J, Rottenberg S, Cantor S, Bartek J, Ray Chaudhuri A, Jonkers J. Loss of nuclear DNA ligase III reverts PARP inhibitor resistance in BRCA1/53BP1 double-deficient cells by exposing ssDNA gaps. Mol Cell. 2021 11 18; 81(22):4692-4708.e9.
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Tekieli T, Yemini E, Nejatbakhsh A, Wang C, Varol E, Fernandez RW, Masoudi N, Paninski L, Hobert O. Visualizing the organization and differentiation of the male-specific nervous system of C. elegans. Development. 2021 09 15; 148(18).