Ontology highlight
ABSTRACT:
INSTRUMENT(S): Q Exactive
ORGANISM(S): Homo Sapiens (human)
TISSUE(S): Erythrocyte, Cell Culture
DISEASE(S): Sickle Cell Anemia
SUBMITTER: Masaya Usui
LAB HEAD: Akihiro Ito
PROVIDER: PXD033536 | Pride | 2023-03-11
REPOSITORIES: Pride
Takase Shohei S Hiroyama Takashi T Shirai Fumiyuki F Maemoto Yuki Y Nakata Akiko A Arata Mayumi M Matsuoka Seiji S Sonoda Takeshi T Niwa Hideaki H Sato Shin S Umehara Takashi T Shirouzu Mikako M Nishigaya Yosuke Y Sumiya Tatsunobu T Hashimoto Noriaki N Namie Ryosuke R Usui Masaya M Ohishi Tomokazu T Ohba Shun-Ichi SI Kawada Manabu M Hayashi Yoshihiro Y Harada Hironori H Yamaguchi Tokio T Shinkai Yoichi Y Nakamura Yukio Y Yoshida Minoru M Ito Akihiro A
Nature communications 20230112 1
Sickle cell disease (SCD) is a heritable disorder caused by β-globin gene mutations. Induction of fetal γ-globin is an established therapeutic strategy. Recently, epigenetic modulators, including G9a inhibitors, have been proposed as therapeutic agents. However, the molecular mechanisms whereby these small molecules reactivate γ-globin remain unclear. Here we report the development of a highly selective and non-genotoxic G9a inhibitor, RK-701. RK-701 treatment induces fetal globin expression bot ...[more]