Therapeutic potential of splice-switching oligonucleotides

by John Bauman, Natee Jearawiriyapaisarn, Ryszard Kole
Published 2018

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

by Natee Jearawiriyapaisarn, Hong M. Moulton, Peter Sazani, Ryszard Kole, Monte S. Willis
Published 2018

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

Downregulation of LAT1 expression suppresses cholangiocarcinoma cell invasion and migration

by Keatdamrong Janpipatkul, Kanoknetr Suksen, Suparerk Borwornpinyo, Natee Jearawiriyapaisarn, Suradej Hongeng, Pawinee Piyachaturawat, Arthit Chairoungdua
Published 2018

The orphan nuclear receptor TR4 regulates erythroid cell proliferation and maturation

by Mary P. Lee, Osamu Tanabe, Lihong Shi, Natee Jearawiriyapaisarn, Daniel Lucas, James Douglas Engel
Published 2018

Molecular analysis of globin gene expression in different thalassaemia disorders: Individual variation of βE pre-mRNA splicing determine disease severity

by Alisa Tubsuwan, Thongperm Munkongdee, Natee Jearawiriyapaisarn, Chanikarn Boonchoy, Pranee Winichagoon, Suthat Fucharoen, Saovaros Svasti
Published 2018

Peptide-morpholino conjugate: A promising therapeutic for duchenne muscular dystrophy

by Hong M. Moulton, Bo Wu, Natee Jearawiriyapaisarn, Peter Sazani, Qi Long Lu, Ryszard Kole
Published 2018

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

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice

by Natee Jearawiriyapaisarn, Hong M. Moulton, Brian Buckley, Jennifer Roberts, Peter Sazani, Suthat Fucharoen, Patrick L. Iversen, Ryszard Kole
Published 2018

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

Inhibition of LSD1 by small molecule inhibitors stimulates fetal hemoglobin synthesis

by Cuong Quang Le, Greggory Myers, Alawi Habara, Natee Jearawiriyapaisarn, George J. Murphy, David H.K. Chui, Martin H. Steinberg, James Douglas Engel, Shuaiying Cui
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

BAP1 regulation of the key adaptor protein NCoR1 is critical for γ-globin gene repression

by Lei Yu, Natee Jearawiriyapaisarn, Mary P. Lee, Tomonori Hosoya, Qingqing Wu, Greggory Myers, Kim Chew Lim, Ryo Kurita, Yukio Nakamura, Anne B. Vojtek, Jean François Rual, James Douglas Engel
Published 2019

Identification of novel γ-globin inducers among all potential erythroid druggable targets

by Lei Yu, Greggory Myers, Emily Schneider, Yu Wang, Raven Mathews, Kim Chew Lim, David Siemieniak, Vi Tang, David Ginsburg, Ginette Balbin-Cuesta, Sharon A. Singh, Pongpon Phuwakanjana, Natee Jearawiriyapaisarn, Rami Khoriaty, James Douglas Engel
Published 2022

An erythroid-to-myeloid cell fate conversion is elicited by LSD1 inactivation

by Lei Yu, Greggory Myers, Chia Jui Ku, Emily Schneider, Yu Wang, Sharon A. Singh, Natee Jearawiriyapaisarn, Andrew White, Takashi Moriguchi, Rami Khoriaty, Masayuki Yamamoto, Michael G. Rosenfeld, Julien Pedron, John H. Bushweller, Kim Chew Lim, James Douglas Engel
Published 2022