Rewriting the script: gene therapy and genome editing for von Willebrand Disease
In recent years gene therapy has emerged as a powerful technology for treatment of a large variety of inherited disorders. With the FDA approval of in vivo gene therapy of hemophilia A and B using AAV-mediated transgene delivery to hepatocytes, the path towards a new treatment era seemed paved. Also...
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| Autori principali: | , , , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Frontiers Media S.A.
2025-09-01
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| Serie: | Frontiers in Genome Editing |
| Soggetti: | |
| Accesso online: | https://www.frontiersin.org/articles/10.3389/fgeed.2025.1620438/full |
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