Gene therapy sickle cell disease

Lebensburger, J. and Persons, D. A. (2008). Progress toward safe and effective gene therapy for beta-thalassemia and sickle cell disease. Curr. Opin. Drug Discov. Devel. 11 225-232. 

Pawliuk R, Westerman KA, Fabry ME, Payen E, Tighe R, et al. 2001. Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294 2368-71... 

Currently, there is stUl a gap for the potential of gene therapy to be fulfilled. Gene therapy clinical trials have been conducted for diseases such as severe combined immunodeficiency disease (SCID, bubble baby syndrome), sickle cell anemia, cystic fibrosis, familial hypercholesterolemia, and Gaucher disease. 

Initially, the focus of gene therapy was for the treatment of inherited disorders such as cystic fibrosis, sickle cell anemia, hemophilia, and adenosine deaminase deficiency. Gene therapy trials were later expanded to include patients with acquired diseases such as cancer and heart disease. 

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