Publications [#99841] of Bruce A Sullenger

Papers Published

1. N Lan, RP Howrey, SW Lee, CA Smith, BA Sullenger, Ribozyme-mediated repair of sickle beta-globin mRNAs in erythrocyte precursors., Science (New York, N.Y.), vol. 280 no. 5369 (June, 1998), pp. 1593-6, ISSN 0036-8075
   (last updated on 2013/05/16)

   Abstract:
   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 beta-globin transcripts in erythrocyte precursors derived from peripheral blood from individuals with sickle cell disease. Sickle beta-globin transcripts were converted into messenger RNAs encoding the anti-sickling protein gamma-globin. These results suggest that RNA repair may become a useful approach in the treatment of genetic disorders.

   Keywords:
   Anemia, Sickle Cell • Cloning, Molecular • Erythroid Precursor Cells • Exons • Fetal Blood • Genetic Therapy • Globins • Humans • Mutation • Polymerase Chain Reaction • RNA Splicing* • RNA, Catalytic • RNA, Messenger • Transfection • Uridine • blood* • chemistry • genetics • genetics* • metabolism • metabolism* • therapy