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2025 21
2026 15

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30 results

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Page 1
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
Reiss UM, Davidoff AM, Tuddenham EGD, Chowdary P, McIntosh J, Muczynski V, Journou M, Simini G, Ireland L, Mohamed S, Riddell A, Pie AJ, Hall A, Quaglia A, Mangles S, Mahlangu J, Haley K, Recht M, Shen YM, Halka KG, Fortner G, Morton CL, Gu Z, Hayden RT, Neufeld EJ, Okhomina VI, Kang G, Nathwani AC. Reiss UM, et al. N Engl J Med. 2025 Jun 12;392(22):2226-2234. doi: 10.1056/NEJMoa2414783. N Engl J Med. 2025. PMID: 40499172 Free PMC article. Clinical Trial.
Cardiotropic AAV gene therapy for heart failure: a phase 1 trial.
Henry TD, Chung ES, Alvisi M, Sethna F, Murray DR, Traverse JH, Roessig L, Roberts L, Reddy S, Chen Y, Ozkan TG, Webb S, Mittal M, Ervin L, Sadek H, Mikhail S, Haghighi K, Jiang C, Samulski RJ, Kranias EG, Tretiakova AP, Hajjar RJ. Henry TD, et al. Nat Med. 2025 Nov;31(11):3845-3852. doi: 10.1038/s41591-025-04011-z. Epub 2025 Oct 21. Nat Med. 2025. PMID: 41120766 Free PMC article. Clinical Trial.
Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.
Sharma A, Locatelli F, Bhatia M, Molinari L, Mapara MY, Liem RI, Dedeken L, Wall D, Eckrich MJ, Kuo KHM, Smith W, Imren S, Kohli P, Li N, Liu T, Rubin J, Hobbs W, Grupp SA, Frangoul H. Sharma A, et al. Blood Adv. 2025 Dec 23;9(24):6481-6490. doi: 10.1182/bloodadvances.2025016701. Blood Adv. 2025. PMID: 40857358 Free PMC article. Clinical Trial.
AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial.
Butterfield RJ, Shieh PB, Li H, Binks M, McDonnell TG, Ryan KA, Delnomdedieu M, Belluscio BA, Neelakantan S, Levy DI, Schwartz PF, Smith EC. Butterfield RJ, et al. Nat Med. 2025 Aug;31(8):2712-2721. doi: 10.1038/s41591-025-03750-3. Epub 2025 Jun 27. Nat Med. 2025. PMID: 40579547 Free PMC article. Clinical Trial.
Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.
Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilić A, Yang L, van der Pol WL. Kwon JM, et al. Nat Med. 2026 Feb;32(2):488-493. doi: 10.1038/s41591-025-04119-2. Epub 2025 Dec 8. Nat Med. 2026. PMID: 41360995 Free PMC article. Clinical Trial.
Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial.
Eichler F, Cataltepe OI, Daci R, Puri AS, Taghian T, Jiang X, Shazeeb MS, Kuhn A, Hader A, Celik H, Vardar Z, Lewis CJ, Artinian R, Nagy A, Vachha B, Thompson R, Gallagher T, Bateman S, Parzych J, Spanakis SG, Vaughn TA, Pier K, De Boever E, Abbott MA, D Ambrosio E, Kokoski D, Blackwood M, Drummond E, Ratai EM, Townsend EL, McLaughlin H, Tifft CJ, Keeler AM, Sena-Esteves M, Gray-Edwards HL, Flotte TR. Eichler F, et al. Nat Med. 2025 Sep;31(9):2927-2935. doi: 10.1038/s41591-025-03822-4. Epub 2025 Aug 15. Nat Med. 2025. PMID: 40817303 Free PMC article. Clinical Trial.
Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial.
Proud CM, Vũ DC, Wilmshurst JM, Sanmaneechai O, Gulati S, Xiong H, Moreno HC, Tay SKH, Thong MK, Born AP, Banzzatto Ortega A, Jong YJ, Al-Muhaizea MA, Lee AW, Visootsak J, Tauscher-Wisniewski S, Alecu I, Parlikar R, Finkel RS; STEER Study Group. Proud CM, et al. Nat Med. 2026 Feb;32(2):481-487. doi: 10.1038/s41591-025-04103-w. Epub 2025 Dec 8. Nat Med. 2026. PMID: 41360993 Free PMC article. Clinical Trial.
Hematopoietic Stem-Cell Gene Therapy for Cystinosis.
Barshop BA, Ball ED, Benador N, Trauner D, Phillips S, Dohil R, Afshari NA, Roy S, Campo Fernandes B, Kohn D, Shayan K, Everett JK, Bushman FD, Midgley J, Liang H, Sawyers A, Gangoiti JA, Panchal M, Ahmed I, Cherqui S. Barshop BA, et al. N Engl J Med. 2026 Feb 19;394(8):753-762. doi: 10.1056/NEJMoa2506431. N Engl J Med. 2026. PMID: 41707137 Free PMC article. Clinical Trial.
Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials.
Xue F, Ju M, Zhu T, Zhou Z, Sun J, Yang L, Yan Z, Zhou H, Du X, Zheng C, Zheng J, Wu X, Du Z, Jiang W, Yang C, Xiao X, Liu W, Yang R, Zhang L. Xue F, et al. Nat Med. 2026 Jan;32(1):93-102. doi: 10.1038/s41591-025-04012-y. Epub 2025 Nov 20. Nat Med. 2026. PMID: 41266685 Free PMC article. Clinical Trial.
AAV9 Gene Therapy in Type II GM1 Gangliosidosis - A Phase 1-2 Trial.
Lewis CJ, D'Souza P, Johnston JM, Acosta MT, Farmer C, Baker EH, Crowell A, Mojica Y, Ashraf S, Joseph L, Vézina G, Quezado Z, Yousef MH, Vardar Z, Shazeeb MS, Corti M, Blackwood M, Coleman K, Batista R, Thurm A, De Boever E, Gahl WA, Byrne BJ, Flotte TR, Jiang X, Gross AL, Keeler AM, Gray-Edwards H, Martin DR, Sena-Esteves M, Tifft CJ. Lewis CJ, et al. N Engl J Med. 2026 Mar 26;394(12):1184-1194. doi: 10.1056/NEJMoa2510935. Epub 2026 Feb 6. N Engl J Med. 2026. PMID: 41665410 Free PMC article. Clinical Trial.
30 results