Targeting Genetic Modifiers of HBG Gene Expression in Sickle Cell Disease: The miRNA Option.

ABSTRACT: Sickle cell disease (SCD) is one of the most common inherited hemoglobinopathy disorders that affects millions of people worldwide. Reactivation of HBG (HBG1, HBG2) gene expression and induction of fetal hemoglobin (HbF) is an important therapeutic strategy for ameliorating the clinical symptoms and severity of SCD. Hydroxyurea is the only US FDA-approved drug with proven efficacy to induce HbF in SCD patients, yet serious complications have been associated with its use. Over the last three decades, numerous additional pharmacological agents that reactivate HBG transcription in vitro have been investigated, but few have proceeded to FDA approval, with the exception of arginine butyrate and decitabine; however, neither drug met the requirements for routine clinical use due to difficulties w

SUBMITTER: Starlard-Davenport A 

PROVIDER: S-EPMC9098152 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature