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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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).
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Benatti HR, Anagnostakou V, Taghian T, Hall EF, Nath S, Heilman CB, Beneduce BM, Leporati A, Raskett C, Epshtein M, King R, Gounis MJ, Malek AM, Gray-Edwards HL. A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP. Mol Ther. 2024 Oct 02; 32(10):3346-3355.
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Harkins AL, Ambegaokar PP, Keeler AM. Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference? Neurotherapeutics. 2024 Jul; 21(4):e00435.