[Progress in Gene Therapy of Sickle Cell Disease Based on Hemoglobin F--Review]

Area of Science:

• Genetics
• Molecular Biology
• Hematology

Background:

• Sickle cell disease (SCD) is a severe single-gene disorder impacting patient lifespan and quality of life.
• Fetal hemoglobin (HbF) induction presents a promising therapeutic avenue for SCD and other beta-hemoglobinopathies.

Purpose of the Study:

• To review the research progress on transcription factors regulating HbF gene expression.
• To explore the application of gene editing technologies for treating SCD.

Main Methods:

• Investigated transcription factors including BCL11A, ZBTB7A, KLF-1, c-MYB, and SOX6.
• Examined gene editing technologies such as CRISPR/Cas9, TALEN, and zinc finger nucleases.

Main Results:

• Identified key transcription factors that control HbF gene expression.
• Demonstrated the potential of gene editing tools in modulating these factors for therapeutic benefit.

Conclusions:

• The identified transcription factors and gene editing technologies provide a strong foundation for developing novel treatments for SCD and beta-thalassemia.
• Further research in this area holds promise for improving therapeutic outcomes for patients with hemoglobin disorders.