AAV-Mediated Gene Transfer for Hemophilia
Author:
Publisher
Wiley
Subject
History and Philosophy of Science,General Biochemistry, Genetics and Molecular Biology,General Neuroscience
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1749-6632.2001.tb11361.x/fullpdf
Reference29 articles.
1. Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease
2. Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B
3. Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B.
4. Expression of human factor IX in mice after injection of genetically modified myoblasts.
5. Posttranslational modifications of recombinant myotube-synthesized human factor IX
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2. Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release;Molecular Therapy - Methods & Clinical Development;2020-06
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