Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B
Author:
Affiliation:
1. Department of Human and Molecular Genetics, Baylor College of Medicine, Houston, TX 77030.
2. Special Coagulation Laboratory, Texas Children's Hospital, Houston, TX 77030.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.liebertpub.com/doi/pdf/10.1089/hum.2008.084
Reference18 articles.
1. Cell‐specific expression of cytosolic phosphoenolpyruvate carboxykinase in transgenic mice
2. Acute Toxicity After High-Dose Systemic Injection of Helper-Dependent Adenoviral Vectors into Nonhuman Primates
3. Sustained Phenotypic Correction of Canine Hemophilia B After Systemic Administration of Helper-Dependent Adenoviral Vector
4. Increased Hepatic Transduction with Reduced Systemic Dissemination and Proinflammatory Cytokines Following Hydrodynamic Injection of Helper-Dependent Adenoviral Vectors
5. Changing Residue 338 in Human Factor IX from Arginine to Alanine Causes an Increase in Catalytic Activity
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1. Gene therapy in PIDs, hemoglobin, ocular, neurodegenerative, and hemophilia B disorders;Open Life Sciences;2021-01-01
2. Systemic delivery of factor IX messenger RNA for protein replacement therapy;Proceedings of the National Academy of Sciences;2017-02-15
3. Clinical development of gene therapy: results and lessons from recent successes;Molecular Therapy - Methods & Clinical Development;2016
4. Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial;Human Gene Therapy;2015-02
5. Gene Therapy: Molecular Engineering of Factor VIII and Factor IX;Textbook of Hemophilia;2014-04-24
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