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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 targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Cancers arise due to mutations in genes involved in the regulation of cell division, which leads to unrestricted cell proliferation. Modern science and medicine have made great strides in the understanding and treatment of cancer, including eradicating cancer in some patients. However, there is still no cure for cancer. This is largely due to the fact that cancer is a large group of many diseases.
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Updated: May 1, 2026

Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
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Genome Editing Technologies for Cancer Therapy.

Gautham Chengizkhan1,2, Arumugam Rajavelu3, Sridhar Muthusami4,5

  • 1Departments of Otolaryngology - Head and Neck Surgery, The University of Oklahoma Health Sciences Center, 800 Research Parkway, Oklahoma City, OK 73104, USA.

Current Gene Therapy
|April 30, 2026
PubMed
Summary
This summary is machine-generated.

Genome editing technologies like CRISPR/CAS9 offer new ways to fight cancer by precisely targeting genes. These advanced tools hold promise for developing more effective precision oncology treatments and improving patient outcomes.

Keywords:
CRISPR/CAS9CancerTALENsgenome editingmeganucleasesprecision medicinezinc finger nucleases (ZFNs)

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Area of Science:

  • Molecular Biology
  • Genetics
  • Oncology

Background:

  • Cancer is a leading global cause of death, with conventional therapies facing limitations like drug resistance and relapse.
  • The complex nature of cancer necessitates innovative therapeutic strategies beyond traditional methods.

Purpose of the Study:

  • To review the evolution and application of genome editing technologies for targeted cancer therapy.
  • To highlight the potential of these tools in revolutionizing precision oncology.

Main Methods:

  • Review of key genome editing platforms: meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/CAS9.
  • Discussion of gene modification strategies including oncogene disruption and tumor suppressor gene repair.

Main Results:

  • Genome editing tools have advanced from early meganucleases to highly efficient RNA-guided CRISPR/CAS9 systems.
  • These technologies enable precise manipulation of genes involved in cancer progression, resistance, and metastasis.

Conclusions:

  • Genome editing technologies show significant translational potential for cancer treatment.
  • Advancements in delivery systems and gene repair enhance therapeutic prospects, paving the way for precision oncology.