Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β^iVS2-654-Thalassemic Erythroid Progenitor Cells

• Main Authors: Annette D'Arqom, Tiwaporn Nualkaew, Natee Jearawiriyapaisarn, Ryszard Kole, Saovaros Svasti
• Other Authors: Universitas Airlangga
• Format: Article
• Published: 2022
• Subjects: Biochemistry, Genetics and Molecular Biology
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/76199
• Institution: Mahidol University

Restoration of correct splicing of βIVS2-654-globin pre-mRNA was previously accomplished in erythroid cells from β-thalassemia/HbE patients by an engineered U7 small nuclear RNA (snRNA) that carried a sequence targeted to the cryptic branch point and an exonic splicing enhancer, U7.BP+623 snRNA. In...