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- Title
Gene therapy: Genie in a vector.
- Authors
Gould, Julie
- Abstract
The article discusses challenges faced by researchers trying to repair faulty genes causing haemophilia especially in children. Treatments include the infusion of a functional factor IX gene along with DNA to control clotting factor, and a form of gene therapy called genome editing. Research by David Wilcox at the Children's Hospital of Wisconsin Research Institute in Milwaukee, to release functioning factor VIII, a blood clotting protein that helps blood clot is presented.
- Subjects
HEMOPHILIA; HEMOPHILIA in children; BLOOD coagulation factor VIII; GENOME editing; WILCOX, David; BLOOD coagulation factor VIII antibodies; BLOOD coagulation factor IX; GENETICS; GENE therapy; THERAPEUTICS
- Publication
Nature, 2014, Vol 515, Issue s7528, pS160
- ISSN
0028-0836
- Publication type
Article
- DOI
10.1038/515S160a