Ribozyme-mediated repair of sickle beta-globin mRNAs in erythrocyte precursors.

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Lan, N; Howrey, RP; Lee, SW; Smith, CA; Sullenger, BA

Published Date

  • June 5, 1998

Published In

Volume / Issue

  • 280 / 5369

Start / End Page

  • 1593 - 1596

PubMed ID

  • 9616120

International Standard Serial Number (ISSN)

  • 0036-8075

Digital Object Identifier (DOI)

  • 10.1126/science.280.5369.1593


  • eng

Conference Location

  • United States