Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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Roger AL, El Haddad L, Huston ML, Kehoe S, Le D, Khan M, Scarrow E, Gonzalez T, Benkert A, Asokan A, ElMallah MK. GAA replacement improves respiratory muscle, neural, and alveolar pathology in the pompe mouse. Respir Physiol Neurobiol. 2025 Jul; 335:104433.
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Belgrad J, McConnell E, Leonard S, Nolen N, Lauffer MC, Watts JK, Yu T, Yan WX, Aartsma-Rus A. The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing. Nucleic Acids Res. 2025 Apr 22; 53(8).
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Keeler AM, Zhan W, Ram S, Fitzgerald KA, Gao G. The curious case of AAV immunology. Mol Ther. 2025 May 07; 33(5):1946-1965.
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Wang D, Stevens G, Flotte TR. Gene therapy then and now: A look back at changes in the field over the past 25 years. Mol Ther. 2025 May 07; 33(5):1889-1902.
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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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Chaugule S, Constantinou CK, John AA, Micha D, Eekhoff M, Gravallese E, Gao G, Shim JH. Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations. Hum Gene Ther. 2025 Mar; 36(5-6):597-617.
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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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Laforet GA. Thrombotic Microangiopathy Associated with Systemic Adeno-Associated Virus Gene Transfer: Review of Reported Cases. Hum Gene Ther. 2025 Feb; 36(3-4):64-76.
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Banou L, Sarrafpour S, Teng CC, Liu J. Ocular Gene Therapy: An Overview of Viral Vectors, Immune Responses, and Future Directions. Yale J Biol Med. 2024 12; 97(4):491-503.
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Aimo A, Olivotto I, Todiere G, Barison A, Panichella G, Fiuzat M, Linde C, Lakdawala NK, Desai M, Zannad F, Maron MS. Endpoint Selection in Randomized Clinical Trials for Hypertrophic Cardiomyopathy. JACC Heart Fail. 2025 Feb; 13(2):200-212.