Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders.
Blood Rev
; 63: 101137, 2024 01.
Article
en En
| MEDLINE
| ID: mdl-37919142
ABSTRACT
Gene modification of haematopoietic stem cells (HSCs) is a potentially curative approach to sickle cell disease (SCD) and offers hope for patients who are not eligible for allogeneic HSC transplantation. Current approaches require in vitro manipulation of healthy autologous HSC prior to their transplantation. However, the health and integrity of HSCs may be compromised by a variety of disease processes in SCD, and challenges have emerged in the clinical trials of gene therapy. There is also concern about increased susceptibility to haematological malignancies during long-term follow up of patients, and this raises questions about genomic stability in the stem cell compartment. In this review, we evaluate the evidence for HSC deficits in SCD and then discuss their potential causation. Finally, we suggest several questions which need to be addressed in order to progress with successful HSC manipulation for gene therapy in SCD.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Trasplante de Células Madre Hematopoyéticas
/
Enfermedades Hematológicas
/
Anemia de Células Falciformes
Límite:
Humans
Idioma:
En
Revista:
Blood Rev
Asunto de la revista:
HEMATOLOGIA
Año:
2024
Tipo del documento:
Article