Gene therapy in an era of emerging treatment options for hemophilia B
Open accessAbstract
Factor IX deficiency (hemophilia B) is less common than factor VIII deficiency (hemophilia A), and innovations in therapy for hemophilia B have generally lagged behind those for hemophilia A. Recently, the first sustained correction of the hemophilia bleeding phenotype by clotting factor gene therapy has been described using recombinant adeno-associated virus (AAV) to deliver factor IX. Despite this success, many individuals with hemophilia B, including children, men with active hepatitis, and individuals who have pre-existing natural immunity to AAV, are not eligible for the current iteration of hemophilia B gene therapy. In addition, recent advances in recombinant factor IX protein engineering have led some hemophilia treaters to reconsider the urgency of genetic cure. Current clinical and preclinical approaches to advancing AAV-based and alternative approaches to factor IX gene therapy are considered in the context of current demographics and treatment of the hemophilia B population.
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Using steroids did not prevent gradual loss of transgene.
“Multiple clinical studies showed that using steroids did not prevent the gradual loss of transgene [211–213].”
From Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer (2023) · cites this paper as [211]
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- Authors
- Monahan, P. E.
- Affiliations
- University of North Carolina at Chapel Hill (US)
- Corresponding author
- Monahan, P.E.
- ORCID
- Monahan, P.E.
- Venue
- Journal of Thrombosis and Haemostasis
- Publisher
- Elsevier BV
- Published
- 2015-06
- Volume
- 13
- Pages
- S151-S160
- ISSN
- 1538-7836 (ISSN-L)
- PMC
- PMC4712690
- OpenAlex
- W1567796113
- Cited by
- 31
- References
- 58
- Citations by year
- 2025: 1 · 2024: 2 · 2023: 2 · 2022: 1 · 2021: 4 · 2020: 2 · 2019: 4 · 2018: 4 · 2017: 4 · 2016: 4 · 2015: 3
- FWCI
- 0.75
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Journal of Thrombosis and Haemostasis (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Carolina Digital Repository (University of North Carolina at Chapel Hill) (submittedVersion)
- UNC Libraries
- Funders
- NIH NHLBI (1RC3HL103396, P01-HL66973, P01-HL112761)
- Asklepios
- Novo Nordisk
- Baxter Healthcare
- Pfizer
- Prolor
- Topics
- Health Sciences › Medicine › Hematology › Hemophilia Treatment and Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Hematology › Platelet Disorders and Treatments
- Keywords
- hemophilia B, gene therapy, adeno-associated virus vectors, recombinant factor IX, treatment eligibility, children, hepatitis C
- MeSH terms
- Dependovirus, Animals, Hemophilia B, Factor IX, Genetic Vectors, Hemostatics, Humans, Male, Recombinant Proteins, Genetic Therapy, Lentivirus, Treatment Outcome, Gene Transfer Techniques, Genetic Predisposition to Disease
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