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Related Concept Videos

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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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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CIRCLE-Seq for Interrogation of Off-Target Gene Editing
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Genome Editing Techniques and Their Therapeutic Applications.

M P Calos1

  • 1Department of Genetics, Stanford University School of Medicine, Stanford, California, USA.

Clinical Pharmacology and Therapeutics
|October 27, 2016
PubMed
Summary

Advances in genome editing technologies offer new therapeutic strategies for genetic diseases and cancer. These DNA editing methods enable precise modifications for potential cures.

Area of Science:

  • Genetics and Molecular Biology
  • Biotechnology
  • Therapeutic Development

Background:

  • Genetic engineering techniques are rapidly advancing.
  • DNA sequence modification offers potential treatments for genetic disorders and cancers.
  • Genome editing tools are crucial for gene and cell therapy development.

Purpose of the Study:

  • To review the expanding landscape of genome editing techniques.
  • To explore the therapeutic applications of these DNA editing methods.
  • To highlight the potential of genome editing in treating diseases.

Main Methods:

  • Review of various genome editing approaches.
  • Analysis of techniques including double-strand break creation, gene addition via retroviruses/transposons, recombinase-mediated editing, and base repair.

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  • Examination of applications in gene and cell therapy.

    • Main Results:

    • Genome editing encompasses diverse methods for DNA modification.
    • Techniques include targeted double-strand breaks, gene addition, and base repair.
    • These methods are being applied to various therapeutic targets.

    Conclusions:

    • Genome editing technologies are continuously evolving.
    • These advancements are creating new therapeutic possibilities.
    • The field holds significant promise for future medical interventions.