Progress in Gene Therapy of Sickle Cell Disease Based on Hemoglobin F--Review / 中国实验血液学杂志
Journal of Experimental Hematology
;
(6): 643-647, 2021.
Artigo
em Chinês
| WPRIM
| ID: wpr-880127
ABSTRACT
Sickle cell disease (SCD) is a single gene genetic disease, which seriously threatens the life span and quality of patients. On the basis of the pathogenesis of SCD and the alternative therapy based on fetal hemoglobin F (HbF), the research progress of transcription factors involved in the regulation of HbF gene expression, such as BCL11A, ZBTB7A, KLF-1, c-MYB and SOX6, as well as the application of CRISPR / Cas9, TALEN, zinc finger nuclease and other gene editing technologies in this field has been made, providing a solid theoretical basis for the exploration of new treatment schemes for β- like hemoglobin diseases, such as sickle cell disease and β- thalassemia.
Texto completo:
DisponíveL
Índice:
WPRIM (Pacífico Ocidental)
Assunto principal:
Proteínas Repressoras
/
Fatores de Transcrição
/
Hemoglobina Fetal
/
Terapia Genética
/
Linhagem Celular Tumoral
/
Proteínas de Ligação a DNA
/
Anemia Falciforme
Limite:
Humanos
Idioma:
Chinês
Revista:
Journal of Experimental Hematology
Ano de publicação:
2021
Tipo de documento:
Artigo
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