Work

Treating sickle cell disease by targeting HbS polymerization

Year: 2017

Type: review

Abstract: Although the root cause of sickle cell disease is the polymerization of hemoglobin S (HbS) to form fibers that make red cells less flexible, most drugs currently being assessed in clinical trials are targeting the downstream sequelae of this primary event. Less attention has been devoted to investigation of the multiple ways in which fiber formation can be inhibited. In this article, we describe the molecular rationale for 5 distinct approaches to inhibiting polymerization and also discuss progress with the few antipolymerization drugs currently in clinical trials.

Source: Blood

DOI: 10.1182/blood-2017-02-765891

Authors: William A. Eaton, H. Franklin Bunn

Institutions: National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, Brigham and Women's Hospital, Harvard University

Language: English

Topic: Hemoglobinopathies and Related Disorders

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: bronze

Funders: National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases