Current state of gene therapy in sickle cell disease.
Vox Sang
; 119(6): 521-528, 2024 Jun.
Article
en En
| MEDLINE
| ID: mdl-38487952
ABSTRACT
Sickle cell disease (SCD) is a type of hemoglobinopathy due to an autosomal recessive genetic defect, causing significant red cell sickling, multi-organ damage and long-term severe morbidities. Due to its complicated care and the impact on quality of life, a curative treatment for SCD is highly desirable. In recent years, gene therapy is emerging as a curative option for SCD, where autologous haematopoietic stem cells are collected from SCD patients and genetically modified ex vivo to reduce its sickling tendency before reinfusion. Although still largely investigational, a limited number of gene therapy options have been recently granted approval for SCD patients. Published data are still currently limited, but early studies have so far demonstrated the intended outcomes of less vaso-occlusive crisis and haemolysis. Nonetheless, despite its curative potential, larger clinical trials and longer follow-up period are still necessary to evaluate the safety of this treatment option, especially the risk of unintended genetic modifications. Furthermore, SCD patients frequently have limited access to specialty care; hence, the issues of affordability and accessibility to SCD gene therapy must also be addressed for it to benefit the appropriate patient population.
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Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Anemia de Células Falciformes
Límite:
Humans
Idioma:
En
Revista:
Vox Sang
Año:
2024
Tipo del documento:
Article
País de afiliación:
Estados Unidos