Ribozyme-Mediated Repair of Sickle -Globin mRNAs in Erythrocyte Precursors

摘要: Sickle cell anemia is the most common heritable hematological disease, yet no curative treatment exists for this disorder. Moreover, intricacies of globin gene expression have made development treatments hemoglobinopathies based on therapy difficult. An alternative genetic approach to sickle RNA repair. A trans-splicing group I ribozyme was used alter mutant β-globin transcripts in erythrocyte precursors derived from peripheral blood individuals with disease. were converted into messenger RNAs encoding anti-sickling protein γ-globin. These results suggest that repair may become a useful disorders.