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

by Annette D'Arqom, Tiwaporn Nualkaew, Natee Jearawiriyapaisarn, Ryszard Kole, Saovaros Svasti
Published 2022

Lysine-specific histone demethylase 1 inhibition enhances robust fetal hemoglobin induction in human β⁰-thalassemia/hemoglobin E erythroid cells

by Woratree Kaewsakulthong, Phitchapa Pongpaksupasin, Tiwaporn Nualkaew, Suradej Hongeng, Suthat Fucharoen, Natee Jearawiriyapaisarn, Orapan Sripichai
Published 2022

Engineered U7 snRNA mediates sustained splicing correction in erythroid cells from β-thalassemia/HbE patients

by Sarinthip Preedagasamzin, Tiwaporn Nualkaew, Tanjitti Pongrujikorn, Natini Jinawath, Ryszard Kole, Suthat Fucharoen, Natee Jearawiriyapaisarn, Saovaros Svasti
Published 2019

Development of DNA controls for detection of β-thalassemia mutations commonly found in Asian

by Thongperm Munkongdee, Tiwaporn Nualkaew, Nattrika Buasuwan, Nurmeeha Hinna, Kittiphong Paiboonsukwong, Orapan Sripichai, Saovaros Svasti, Pranee Winichagoon, Suthat Fucharoen, Natee Jearawiriyapaisarn
Published 2020

UNC0638 induces high levels of fetal hemoglobin expression in β-thalassemia/HbE erythroid progenitor cells

by Tiwaporn Nualkaew, Pinyaphat Khamphikham, Phitchapa Pongpaksupasin, Woratree Kaewsakulthong, Duantida Songdej, Kittiphong Paiboonsukwong, Orapan Sripichai, James Douglas Engel, Suradej Hongeng, Suthat Fucharoen, Natee Jearawiriyapaisarn
Published 2020

High-level induction of fetal haemoglobin by pomalidomide in β-thalassaemia/HbE erythroid progenitor cells

by Pinyaphat Khamphikham, Tiwaporn Nualkaew, Phitchapa Pongpaksupasin, Woratree Kaewsakulthong, Duantida Songdej, Kittiphong Paiboonsukwong, James D. Engel, Suradej Hongeng, Suthat Fucharoen, Orapan Sripichai, Natee Jearawiriyapaisarn
Published 2020

Enhancement of β-globin gene expression in thalassemic IVS2-654 induced pluripotent stem cell-derived erythroid cells by modified U7 snRNA

by Phetcharat Phanthong, Suparerk Borwornpinyo, Narisorn Kitiyanant, Natee Jearawiriyapaisarn, Lalana Nuntakarn, Jirawat Saetan, Tiwaporn Nualkaew, Khanit Sa-Ngiamsuntorn, Usanarat Anurathapan, Andras Dinnyes, Yindee Kitiyanant, Suradej Hongeng
Published 2018

Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia

by Tiwaporn Nualkaew, Karine Sii-Felice, Marie Giorgi, Bradley McColl, Julie Gouzil, Astrid Glaser, Hsiao P.J. Voon, Hsin Y. Tee, George Grigoriadis, Saovaros Svasti, Suthat Fucharoen, Suradej Hongeng, Philippe Leboulch, Emmanuel Payen, Jim Vadolas
Published 2022