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Gene Editing in Clinical Practice: Where are We?

Rama Devi Mittal1

  • 1Department of Urology and Renal Transplantation, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Raebareli Road, Lucknow, Uttar Pradesh 226014 India.

Indian Journal of Clinical Biochemistry : IJCB
|February 8, 2019
PubMed
Summary
This summary is machine-generated.

The CRISPR/Cas9 gene editing system offers precise genetic modification capabilities, revolutionizing disease research and treatment development. Ongoing advancements aim to improve its precision, delivery, and safety for future therapeutic applications.

Keywords:
CASPR Cas9 systemsEthicsGene targetingGene therapy embryoGenome editingMolecular scissorsNucleasesPalindromic repeatsPotential pitfalls

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-associated protein 9 (CRISPR/Cas9) is a powerful gene editing tool.
  • Its simplicity, cost-effectiveness, and design flexibility have led to widespread adoption in research.
  • CRISPR/Cas9 leverages bacterial defense mechanisms for precise DNA targeting and modification.

Purpose of the Study:

  • To review the capabilities and applications of the CRISPR/Cas9 system.
  • To discuss its potential for correcting genetic mutations and generating disease models.
  • To highlight areas needing improvement, including precision, delivery, and ethical considerations.

Main Methods:

  • CRISPR/Cas9 system utilizes a guide RNA to direct the Cas9 enzyme (molecular scissors) to specific DNA sequences.
  • Double-strand breaks are introduced and repaired via homologous recombination or non-homologous end joining (NHEJ).
  • NHEJ can lead to gene knockouts through indel formation, while homologous recombination allows for precise edits or gene insertions.

Main Results:

  • CRISPR/Cas9 enables precise gene editing, including gene knockouts, insertions, and single-letter modifications.
  • The system has been successfully used to create genetic disease models for research.
  • Potential for therapeutic applications in correcting deleterious mutations is significant.

Conclusions:

  • CRISPR/Cas9 technology is revolutionizing genetic research and holds immense promise for treating genetic diseases.
  • Further research is crucial to address challenges in precision, delivery, control, and biosafety.
  • Ethical considerations and safeguards are paramount for responsible development and application.