FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B.
prospective_cohort · Level II
Where this comes from
- Record sourced from PubMed, PMID 29245009.
- Also identified by DOI 10.1016/j.cell.2017.11.049.
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Abstract
A phase 1/2 clinical trial of AAV-mediated gene therapy in patients with hemophilia B using an enhanced specific activity factor IX (FIX) transgene reports sustained levels of FIX levels, leading to the near elimination of bleeding for more than a year and without serious adverse side effects. These results are the best outcome to date for hemophilia gene therapy.
Medical subject headings
- Factor IX
- Hemophilia B