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Related Experiment Video

Updated: Feb 3, 2026

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Emerging Genetic Therapy for Sickle Cell Disease.

Stuart H Orkin1,2, Daniel E Bauer1

  • 1Dana Farber/Boston Children's Cancer and Blood Disorders Center, Department of Pediatrics, Harvard Medical School, Boston, Massachusetts 02115, USA;

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|October 26, 2018
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Summary

Genetic insights into sickle cell disease (SCD) are paving the way for new therapies. Advances in gene editing and gene therapy offer potential cures by targeting fetal hemoglobin production and correcting the SCD mutation.

Keywords:
BCL11ACRISPR/Cas9HbFLRF/ZBTB7Afetal hemoglobingene therapysickle cell disease

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Area of Science:

  • Genetics
  • Molecular Biology
  • Hematology

Background:

  • The genetic basis of sickle cell disease (SCD) has been known for over 60 years, but current treatments do not leverage this understanding.
  • Recent scientific discoveries offer new hope for improved and potentially curative treatments for SCD.

Purpose of the Study:

  • To explore recent advances in understanding the genetic regulation of fetal hemoglobin and advancements in gene-based therapies for sickle cell disease.
  • To highlight the potential of gene editing and gene therapy for curing SCD.

Main Methods:

  • Identification and characterization of transcription factors (BCL11A, LRF/ZBTB7A) involved in silencing fetal (γ-) globin gene expression.
  • Leveraging advances in gene editing technologies and lentiviral gene therapy for therapeutic strategies.

Main Results:

  • Transcription factors BCL11A and LRF/ZBTB7A were identified as key mediators of γ-globin gene silencing at specific promoter recognition sequences.
  • Gene editing and lentiviral gene therapy present diverse opportunities for genetic interventions in SCD.

Conclusions:

  • Genetic strategies, including gene addition, gene editing to correct mutations, and enhancing fetal hemoglobin, show promise for curing SCD.
  • Upcoming clinical trials are expected to evaluate the efficacy and safety of these genetic approaches for the definitive treatment of SCD.