Associated Papers
Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B.
Reiss UM et al.
The New England Journal of Medicine, 2025
Splicing dysregulation contributes to the pathogenicity of several F9 exonic point variants.
Katneni UK et al.
Molecular Genetics & Genomic Medicine, 2019
31257730 (PubMed )
Supplement: MGG3-7-e840-s002.xls › F9_Splicing_Data
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC et al.
The New England Journal of Medicine, 2014
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