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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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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Daci R, Gray-Edwards H, Shazeeb MS, Vardar Z, Vachha B, Cataltepe OI, Flotte TR. Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases. Hum Gene Ther. 2024 Nov; 35(21-22):886-895.
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Wang JH, Zhan W, Gallagher TL, Gao G. Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review. Mol Ther. 2024 Dec 04; 32(12):4185-4207.
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Du A, Yang K, Zhou X, Ren L, Liu N, Zhou C, Liang J, Yan N, Gao G, Wang D. Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiency. JCI Insight. 2024 Oct 08; 9(19).
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Mo W, Donahue JK. Gene therapy for atrial fibrillation. J Mol Cell Cardiol. 2024 Nov; 196:84-93.
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Pires-Ferreira D, Reil D, Tang Q, Blackwood M, Gallagher T, Keeler AM, Chichester JA, Vyhnal KK, Lindborg JA, Benson J, Fu D, Flotte TR, Gruntman AM. Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy. Genes (Basel). 2024 Sep 10; 15(9).