New England Journal of Medicine· 2014Q1
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
- 1,257citations
- Q1SCImago
- 2014year
Short summary
A single AAV8 vector infusion in 10 severe hemophilia B patients yielded sustained Factor IX expression (1-6% of normal) for over 3 years, reducing bleeding episodes by >90% in high-dose recipients.
AI-generated from the title and abstract; the full text is not read.
TakeawaysIn the app
Key pointsIn the app
Ask the paperIn the app
The rest is in the Pofolia app
Takeaways, key points and questions to the paper; new summaries every day for your field. Free.
Sign in on the web to openField: Hematology
HematologyMedicine