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Related Concept Videos

CRISPR01:59

CRISPR

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Homologous Recombination02:31

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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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CRISPR and crRNAs02:53

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
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Updated: Jan 12, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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CRISPR and gene editing technologies for bleeding disorders.

Thierry VandenDriessche1, Mathias Janssens2, Marinee K Chuah3

  • 1Department of Gene Therapy and Regenerative Medicine, Faculty of Medicine and Pharmacy, Vrije Universiteit Brussel, Building D, room JD.3.56, Laarbeeklaan 103, Brussels B-1090, Belgium.

Therapeutic Advances in Hematology
|November 3, 2025
PubMed
Summary

Gene editing offers a promising new avenue for treating hemophilia A and B, moving beyond current gene therapies. Preclinical studies show sustained factor production, paving the way for clinical trials.

Keywords:
CRISPRTALENZFNadeno-associatedbase editingfactor IXfactor VIIIhemophiliananoparticles

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Area of Science:

  • Biotechnology
  • Hematology
  • Genetic Engineering

Background:

  • Recent approval of gene therapies for hemophilia A and B marks a significant advancement.
  • Existing therapies require improvements in efficacy, safety, and expression stability, especially for pediatric patients.
  • Gene editing presents a novel strategy to address limitations of conventional gene therapy.

Purpose of the Study:

  • To explore the potential of gene editing technologies for treating hemophilia A and B.
  • To evaluate the efficacy and safety of gene editing strategies in preclinical models.
  • To assess the feasibility of gene editing for sustained factor VIII or IX production.

Main Methods:

  • Utilized zinc finger nucleases, meganucleases, TALENs, and CRISPR technologies for targeted DNA modification.
  • Investigated CRISPR-derived base and prime editors for DSB-independent gene editing.
  • Conducted preclinical studies in patient-derived cells and hemophilia A or B mouse models.

Main Results:

  • Gene editing demonstrated sustained efficacy and production of factor VIII or IX in preclinical models.
  • Next-generation editors (base/prime) offer potential for safer genetic modification without DSBs.
  • Preclinical data supports the advancement of gene editing towards clinical trials for severe hemophilia.

Conclusions:

  • Gene editing holds significant promise for the future treatment of hemophilia.
  • Further research is crucial to address off-target effects, immune responses, and delivery challenges.
  • Successful translation of gene editing to the clinic could revolutionize hemophilia care.