Defining curative endpoints for sickle cell disease in the era of gene therapy and gene editing
Franco Locatelli,
Selim Corbacioglu,
William Hobbs
et al.
Abstract:A growing number of gene therapy‐ and gene editing‐based treatments for patients with sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the underlying cause of SCD, have the potential to provide functional cures, which until now were possible only through allogeneic hematopoietic stem cell transplant. However, as these novel approaches advance from early‐ to late‐stage clinical trials, it is essential to identify physiologically and clinically relevant endpoints that … Show more
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.