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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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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CRISPR01:59

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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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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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Genomics02:02

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Genomics is the science of genomes: it is the study of all the genetic material of an organism. In humans, the genome consists of information carried in 23 pairs of chromosomes in the nucleus, as well as mitochondrial DNA. In genomics, both coding and non-coding DNA is sequenced and analyzed. Genomics allows a better understanding of all living things, their evolution, and their diversity. It has a myriad of uses: for example, to build phylogenetic trees, to improve productivity and...
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The hematopoietic stem cells or HSCs are multipotent, meaning they can differentiate and give rise to all blood and immune cells. HSCs are maintained in the quiescent stage until an external stimulus initiates their differentiation. The multipotent HSCs exist as two heterogeneous populations, long-term repopulating cells (LTRC) and short-term repopulating cells (STRC). The two HSC populations have different surface markers or receptors and are classified based on quiescence and long-term...
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Updated: Feb 5, 2026

Genome Engineering of Primary Human B Cells Using CRISPR/Cas9
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CRISPR/Cas9 genome engineering in hematopoietic cells.

Duran Sürün1, Harald von Melchner2, Frank Schnütgen2

  • 1Department of Molecular Hematology and LOEWE Center for Cell and Gene Therapy, Goethe University Medical School, 60590 Frankfurt am Main, Germany; Medical Systems Biology, UCC, Medical Faculty Carl Gustav Carus, TU Dresden, Fetscherstr. 74, 01307 Dresden, Germany.

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Summary

CRISPR/Cas9 gene editing shows promise for treating blood disorders, HIV, and cancer by precisely modifying hematopoietic cells. This review covers current achievements and limitations in its therapeutic application.

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Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9
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Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models
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Genome Engineering of Primary Human B Cells Using CRISPR/Cas9
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Highly Efficient Gene Disruption of Murine and Human Hematopoietic Progenitor Cells by CRISPR/Cas9
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Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models
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Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Hematology

Background:

  • Advanced genome editing tools enable precise DNA modification.
  • CRISPR/Cas9, derived from prokaryotic immunity, offers simplicity and flexibility.
  • Hematopoietic cells are key targets for genetic therapies.

Purpose of the Study:

  • Review CRISPR/Cas9 gene editing in hematopoietic cells.
  • Highlight potential for ex vivo gene therapy.
  • Discuss applications in monogenic blood disorders, HIV, and cancer.

Main Methods:

  • Literature review of CRISPR/Cas9 applications.
  • Analysis of gene editing efficiency and specificity in hematopoietic cells.
  • Evaluation of therapeutic potential and limitations.

Main Results:

  • CRISPR/Cas9 demonstrates significant potential for targeted gene manipulation in hematopoietic cells.
  • Ex vivo gene therapy approaches show promise for monogenic blood disorders.
  • CRISPR/Cas9 is being explored for HIV and cancer treatment strategies.

Conclusions:

  • CRISPR/Cas9 gene editing is a powerful tool for hematopoietic cell modification.
  • Further research is needed to overcome limitations for widespread clinical use.
  • Ex vivo gene therapy holds significant promise for treating various blood-related diseases and conditions.