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Related Concept Videos

Regulation of Hematopoietic Stem Cells01:01

Regulation of Hematopoietic Stem Cells

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All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...
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Related Experiment Video

Updated: Jul 5, 2025

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 in Hematopoietic Stem Cells.

Jiaoyang Liao1, Yuxuan Wu2

  • 1Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China.

Advances in Experimental Medicine and Biology
|January 16, 2024
PubMed
Summary
This summary is machine-generated.

Gene editing tools are revolutionizing hematopoietic stem cell (HSC) therapies for blood disorders. This review covers gene editing applications in HSCs, clinical advances, and challenges for effective treatments.

Keywords:
ChallengesClinical trialsGene editingHSCPerspectives

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Area of Science:

  • Hematology
  • Molecular Biology
  • Gene Therapy

Background:

  • Hematopoietic stem cells (HSCs) are crucial for blood system function and can be modified ex vivo.
  • Gene editing technologies offer precise genetic modification of cells, including HSCs.

Approach:

  • This review details the evolution of gene editing tools.
  • It explores diverse strategies for applying these tools to hematopoietic stem and progenitor cells (HSPCs).

Key Points:

  • Gene editing in HSPCs is vital for understanding hematopoiesis and developing novel therapies.
  • Significant clinical progress has been made in treating immune and inherited blood disorders using HSPC gene editing.

Conclusions:

  • HSPC gene editing holds immense therapeutic potential for various genetic diseases.
  • Overcoming challenges in clinical translation is key to realizing the full benefits of these advanced therapies.