Gene therapy for hemophilia
Peter J Lenting, Sylvia Fong · 2024
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Description
Peter J Lenting and Sylvia Fong summarize gene therapy for hemophilia in a short overview. Their 2024 piece explains the concept first: a harmless virus delivers a working copy of the clotting factor gene to liver cells. One infusion can then supply the missing factor for years, cutting bleeding episodes.
Long-term studies now report on how well the approach works, how safe it is, and how long the benefit lasts. Therapies for both hemophilia A and B have gained approval in some regions. Most treated patients show a large drop in bleeding and no longer need regular factor replacement. The authors close with future steps: better vectors, stronger expression, and calmer immune reactions could widen who can be treated. The overview suits readers who want the field's state in a few pages.
Catalog record
| CREATOR | Peter J Lenting, Sylvia Fong |
|---|---|
| YEAR | 2024 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 2 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |