Article
Ribozyme-mediated repair of sickle beta-globin mRNAs in erythrocyte precursors.
Science (New York, N.Y.) - 5 Jun 1998
Lan N, Howrey R P, Lee S W, Smith C A, Sullenger B A
Abstract excerpt
Sickle cell anemia is the most common heritable hematological disease, yet no curative treatment exists for this disorder. Moreover, the intricacies of globin gene expression have made the development of treatments for hemoglobinopathies based on gene therapy difficult. An alternative genetic approach to sickle cell therapy is based on RNA repair. A trans-splicing group I ribozyme was used to alter mutant...
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