Potential limits of AAV-based gene therapy with the use of new transgenes expressing factor IX fusion proteins
Author:
Affiliation:
1. EA 4609-Hémostase et Cancer; Université Claude Bernard Lyon I; Lyon France
2. Laboratoire D’hématologie; Hospices Civils de Lyon; Lyon France
3. Research Department of Haematology; University of London Cancer Institute; London UK
Funder
Fondation Groupama pour la Santé - Vaincre les maladies Rares
Publisher
Wiley
Subject
Genetics(clinical),Hematology,General Medicine
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/hae.13651/fullpdf
Reference33 articles.
1. Genetic fusion to albumin improves the pharmacokinetic properties of factor IX;Metzner;Thromb Haemost,2009
2. Prolonged activity of factor IX as a monomeric Fc fusion protein;Peters;Blood,2010
3. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial;Santagostino;Blood,2016
4. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B;Powell;N Engl J Med,2013
5. The major histocompatibility complex-related Fc receptor for IgG (FcRn) binds albumin and prolongs its lifespan;Chaudhury;J Exp Med,2003
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1. Half-Life Extension Enhances Drug Efficacy in Adeno-Associated Virus Delivered Gene Therapy;Engineering;2023-02
2. Gene Therapy For Hemophilia B Using CB 2679d-GT: A Novel Factor IX Variant With Higher Potency Than Factor IX Padua;Blood;2021-03-18
3. Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy;Molecular Therapy - Methods & Clinical Development;2019-03
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