Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced.

ABSTRACT:

Introduction

Sickle cell disease (SCD) is the most common inherited hemoglobinopathy worldwide, and is a life-limiting disease with limited therapeutic options to reduce disease severity. Despite being a monogenic disorder, the clinical phenotypes of SCD are variable, with few reliable predictors of disease severity easily identifying patients where the benefits of curative therapy outweigh the risks. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option, though significant advances in gene therapy raise the promise for additional curative methods. Areas covered: Allogeneic transplantation in SCD has evolved and improved over the last two decades, now offering a standard of care curative option using a human leukocyte antigen (HLA)-matched sibling do

SUBMITTER: Leonard A 

PROVIDER: S-EPMC8459571 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature