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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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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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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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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Updated: Feb 20, 2026

A New Toolkit for Evaluating Gene Functions using Conditional Cas9 Stabilization
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[CRISPR/Cas9 and gene therapy].

Luciana E Giono1

  • 1Instituto de Fisiología, Biología Molecular y Neurociencias (IFIBYNE), CONICET-Universidad de Buenos Aires, Buenos Aires, Argentina.

Medicina
|October 19, 2017
PubMed
Summary
This summary is machine-generated.

CRISPR/Cas9 gene editing technology, adapted from bacteria, offers precise and accessible genome modification for biomedical research and gene therapy. Its revolutionary potential requires careful consideration for safe application.

Keywords:
CRISPR/Cas9gene therapygenome editinghuman embryos

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Area of Science:

  • Biomedical Research
  • Molecular Biology
  • Genetics

Background:

  • Precise genome editing is a key objective in biomedical research.
  • Various genome editing tools have emerged over decades.
  • CRISPR/Cas9, a bacterial defense system, is a prominent tool.

Purpose of the Study:

  • To review the development and impact of genome editing technologies.
  • To highlight the significance of the CRISPR/Cas9 system.
  • To discuss the implications for gene therapy.

Main Methods:

  • Review of existing literature on genome editing techniques.
  • Focus on the CRISPR/Cas9 system's mechanism and applications.
  • Analysis of CRISPR/Cas9's accessibility and potential.

Main Results:

  • CRISPR/Cas9 has revolutionized biomedical sciences due to its precision and efficiency.
  • The system is technically and economically accessible.
  • It represents a significant advancement in gene therapy.

Conclusions:

  • CRISPR/Cas9 technology has transformed biomedical research and holds promise for gene therapy.
  • The widespread adoption and potential of CRISPR/Cas9 necessitate a cautious approach.
  • Further research and ethical considerations are crucial for its responsible application.