Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.
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| Title: | Clinical perspective: Advancing hemophilia treatment through gene therapy approaches. |
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| Authors: | Thornburg CD; National Institutes of Health, National Heart, Lung, and Blood Institute, Division of Blood Diseases and Resources, Bethesda, MD, USA., Pipe SW; Pediatric Hematology-Oncology, University of Michigan, Ann Arbor, MI, USA., Cantore A; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy; Vita-Salute San Raffaele University, Milan, Italy., Unzu C; DNA and RNA Medicine Division, CIMA Universidad de Navarra, Pamplona, Spain., Jones M; Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA., Miesbach WA; Department of Haemostaseology University Hospital Frankfurt, Frankfurt, Germany. Electronic address: miesbach@em.uni-frankfurt.de. |
| Source: | Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2025 Jun 04; Vol. 33 (6), pp. 2350-2362. Date of Electronic Publication: 2025 Apr 21. |
| Publication Type: | Journal Article; Review |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| ISSN: | 1525-0024 |
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| DOI: | 10.1016/j.ymthe.2025.04.023 |
