Below are the most recent publications written about "Genetic Therapy" 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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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 Jan; 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 Dec; 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 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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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 Oct 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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Matesanz SE, Brigatti KW, Young M, Yum SW, Strauss KA. Preemptive dual therapy for children at risk for infantile-onset spinal muscular atrophy. Ann Clin Transl Neurol. 2024 Jul; 11(7):1868-1878.