A scoping review exploring cure definitions and language for inherited hemoglobinopathies.

ABSTRACT:

Purpose

Sickle cell disease and beta thalassemia are some of the first targets for potentially curative cell-based therapies. Currently, bone marrow transplants, stem cell transplants, and gene therapy are being researched and utilized for people living with these hemoglobinopathies. Although these therapies are often described as curative, there is not a clear definition of what cure means for these hemoglobinopathies.

Methods

Five databases were searched for this scoping review. Two reviewers screened each article at the title/abstract and full text levels using Covidence. Articles were included if they were (1) about bone marrow transplants, stem cell transplants, or gene therapy; (2) conditions of focus were sickle cell disease or beta thalassemia; and (3) reported origi

SUBMITTER: Baffoe-Bonnie MS 

PROVIDER: S-EPMC10956708 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature