Article
Engineered U7 snRNA mediates sustained splicing correction in erythroid cells from β-thalassemia/HbE patients.
Biochemical and biophysical research communications - 30 Apr 2018
Preedagasamzin Sarinthip, Nualkaew Tiwaporn, Pongrujikorn Tanjitti, Jinawath Natini, Kole Ryszard, Fucharoen Suthat, Jearawiriyapaisarn Natee, Svasti Saovaros
Abstract excerpt
Repair of a splicing defect of β-globin pre-mRNA harboring hemoglobin E (HbE) mutation was successfully accomplished in erythroid cells from patients with β-thalassemia/HbE disorder by a synthetic splice-switching oligonucleotide (SSO). However, its application is limited by short-term effectiveness and requirement of lifelong periodic administration of SSO, especially for chronic diseases like thalassemias....
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