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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

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Gene editing for sickle cell disease and transfusion dependent thalassemias- A cure within reach

Michael J Eckrich1, Haydar Frangoul2

  • 1From Atrium Health Levine Children's Hospital, Charlotte, NC.

Seminars in Hematology
|April 20, 2023
PubMed

Insights

Gene editing offers a promising new treatment for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TdT). This approach aims to correct genetic defects and restore fetal hemoglobin production, improving patient outcomes.

Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TdT) cause severe health issues and reduced lifespan.
  • Current treatments like allogeneic transplantation are limited by donor availability and complications.
  • Existing gene therapy for TdT shows promise, but new approaches are needed.

Purpose of the Study:

  • To review the potential of gene editing technologies for treating SCD and TdT.
  • To explore how gene editing can induce fetal hemoglobin (HbF) production.
  • To discuss the application of CRISPR/Cas9 in correcting genetic defects in hematopoietic stem cells.

Main Methods:

  • Review of current research on gene editing tools like CRISPR/Cas9.
  • Analysis of molecular pathways controlling erythropoiesis and globin switching.
  • Examination of ex vivo gene therapy approaches for SCD and TdT.

Main Results:

  • Gene editing offers a potential curative approach for SCD and TdT.
  • CRISPR/Cas9 technology enables precise genetic modification of patient-derived cells.
  • Inducing fetal hemoglobin production is a key strategy for therapeutic benefit.

Conclusions:

  • Genome editing presents a novel and exciting therapeutic avenue for SCD and TdT.
  • Advances in understanding erythropoiesis and globin switching inform gene editing strategies.
  • Further research and clinical application of gene editing hold significant promise for these genetic blood disorders.

Keywords:
Allogeneic transplantAutologous transplantCRISPR-Cas9Sickle cell diseaseTransfusion dependent thalassemia

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