Gene therapy, an ongoing revolution
Author:
Affiliation:
1. UNIVERSITÉ PIERRE ET MARIE CURIE
Publisher
American Society of Hematology
Subject
Cell Biology,Hematology,Immunology,Biochemistry
Link
http://ashpublications.org/blood/article-pdf/119/13/2973/1348869/zh801312002973.pdf
Reference11 articles.
1. Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.;Buchlis;Blood,2012
2. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.;Manno;Blood,2003
3. Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy.;Romero;Hum Gene Ther,2004
4. Efficacy of gene therapy for X-linked severe combined immunodeficiency.;Hacein-Bey-Abina;N Engl J Med,2010
5. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.;Manno;Nat Med,2006
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3. Development of Methods for the Selective Measurement of the Single Amino Acid Exchange Variant Coagulation Factor IX Padua;Molecular Therapy - Methods & Clinical Development;2018-09
4. Novel electric power-driven hydrodynamic injection system for gene delivery: safety and efficacy of human factor IX delivery in rats;Gene Therapy;2013-01-24
5. Current World Literature;Current Opinion in Pediatrics;2012-12
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