Sickle cell disease in the era of precision medicine: looking to the future.

ABSTRACT:

Introduction

Sickle cell anemia is a mendelian disease that is noted for the heterogeneity of its clinical expression. Because of this, providing an accurate prognosis has been a longtime quest.

Areas covered

Reviewed are the benefits and shortcomings of testing for the major modulators of the severity of disease, like fetal hemoglobin and α thalassemia, along with studies that have attempted to link genetic variation with sub-phenotypes of disease in a predictive fashion. Induced pluripotent stem cells driven to differentiate into erythroid precursor cells provide another area for potential patient-specific drug testing.

Expert opinion

Fetal hemoglobin is the strongest modulator of sickle cell anemia but simply measuring its blood levels is an insufficient means of f

SUBMITTER: Steinberg MH 

PROVIDER: S-EPMC7531762 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature