Below are the most recent publications written about "Genetic Vectors" by people in Profiles.
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Wang J, Poskitt LE, Gallagher J, Puffenberger EG, Wynn RM, Shishodia G, Chuang DT, Beever J, Hardin DL, Brigatti KW, Baker WC, Gately R, Bertrand S, Rodrigues A, Benatti HR, Taghian T, Hall E, Prestigiacomo R, Liang J, Chen G, Zhou X, Ren L, Liu N, He R, Su Q, Xie J, Jiang Z, Gruntman A, Gray-Edwards H, Gao G, Strauss KA, Wang D. BCKDHA-BCKDHB digenic gene therapy restores metabolic homeostasis in two mouse models and a calf with classic maple syrup urine disease. Sci Transl Med. 2025 Feb 26; 17(787):eads0539.
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Yu-Wai-Man P, Newman NJ, Biousse V, Carelli V, Moster ML, Vignal-Clermont C, Klopstock T, Sadun AA, Sergott RC, Hage R, Degli Esposti S, La Morgia C, Priglinger C, Karanja R, Taiel M, Sahel JA. Five-Year Outcomes of Lenadogene Nolparvovec Gene Therapy in Leber Hereditary Optic Neuropathy. JAMA Ophthalmol. 2025 Feb 01; 143(2):99-108.
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Herre?o-Pach?n AM, Sawamoto K, Stapleton M, Khan S, Piechnik M, ?lvarez JV, Tomatsu S. Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice. Hum Gene Ther. 2024 Dec; 35(23-24):955-968.
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Celik B, Rintz E, Sansanwal N, Khan S, Bigger B, Tomatsu S. Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model. Hum Gene Ther. 2024 Nov; 35(21-22):917-937.
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Lerchenm?ller C, Hastings MH, Rabolli CP, Betge F, Roshan M, Liu LX, Liu X, He? C, Roh JD, Platt C, Bezzerides V, Busch M, Katus HA, Frey N, Most P, Rosenzweig A. CITED4 gene therapy protects against maladaptive cardiac remodeling after ischemia/reperfusion injury in mice. Mol Ther. 2024 Oct 02; 32(10):3683-3694.
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Lau CH, Rouhani MJ, Maughan EF, Orr JC, Kolluri KK, Pearce DR, Haughey EK, Sutton L, Flatau S, Balboa PL, Bageta ML, O'Callaghan C, Smith CM, Janes SM, Hewitt R, Petrof G, Martinez AE, McGrath JA, Butler CR, Hynds RE. Lentiviral expression of wild-type LAMA3A restores cell adhesion in airway basal cells from children with epidermolysis bullosa. Mol Ther. 2024 May 01; 32(5):1497-1509.
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Lu Y, Zhang CX, Rodrigues P, Yang L, Shafer A, Rankovic Z, Fazio F. Development and Validation of a Liquid Chromatography-Tandem Mass Spectrometry Method for Sensitive Analysis of Residual Protein Tat Bh1-101 in Lentiviral Vectors for Gene Therapy. Hum Gene Ther. 2023 11; 34(21-22):1162-1171.
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Fu Q, Lee YS, Green EA, Wang Y, Park SY, Polanco A, Lee KH, Betenbaugh M, McNally D, Yoon S. Design space determination to optimize DNA complexation and full capsid formation in transient rAAV manufacturing. Biotechnol Bioeng. 2023 11; 120(11):3148-3162.
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Vignal-Clermont C, Yu-Wai-Man P, Newman NJ, Carelli V, Moster ML, Biousse V, Subramanian PS, Wang AG, Donahue SP, Leroy BP, Sadun AA, Klopstock T, Sergott RC, Rebolleda Fernandez G, Chwalisz BK, Banik R, Taiel M, Roux M, Sahel JA. Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy. Am J Ophthalmol. 2023 05; 249:108-125.
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Jagram N, Dasgupta I. Principles and practice of virus induced gene silencing for functional genomics in plants. Virus Genes. 2023 Apr; 59(2):173-187.