Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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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 02 01; 143(2):99-108.
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Van Linthout S, Stellos K, Giacca M, Bertero E, Cannata A, Carrier L, Garcia-Pavia P, Ghigo A, Gonz?lez A, Haugaa KH, Imazio M, Lopes LR, Most P, Pollesello P, Schunkert H, Streckfuss-B?meke K, Thum T, Tocchetti CG, Tsch?pe C, van der Meer P, van Rooij E, Metra M, Rosano GMC, Heymans S. State of the art and perspectives of gene therapy in heart failure. A scientific statement of the Heart Failure Association of the ESC, the ESC Council on Cardiovascular Genomics and the ESC Working Group on Myocardial & Pericardial Diseases. Eur J Heart Fail. 2025 01; 27(1):5-25.
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Goldenberg M, Lanzkron S, Pecker LH. Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management. Expert Rev Hematol. 2024 12; 17(12):891-905.
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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 12; 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 11; 35(21-22):917-937.
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Celik B, Leal AF, Tomatsu S. Potential Targeting Mechanisms for Bone-Directed Therapies. Int J Mol Sci. 2024 Jul 30; 25(15).
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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 10 02; 32(10):3683-3694.
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Wheeler S, Mahmoudzadeh R, Randolph J. Treatment for dry age-related macular degeneration: where we stand in 2024. Curr Opin Ophthalmol. 2024 Sep 01; 35(5):359-364.
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Guri-Lamce I, Alrokh Y, Graham C, Maeshima R, Rognoni E, Caley M, Laczmanski L, Hart SL, McGrath JA, Jack?w-Malinowska J. Lipid Nanoparticles Efficiently Deliver the Base Editor ABE8e for COL7A1 Correction in Dystrophic Epidermolysis Bullosa Fibroblasts In?Vitro. J Invest Dermatol. 2024 Oct; 144(10):2314-2317.e3.
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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 05 01; 32(5):1497-1509.