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Current genome editing tools in gene therapy: new approaches to treat cancer

Oleg Shuvalov, Alexey Petukhov, Alexandra Daks

  • 1Head of the Laboratory of Gene Expression Regulation, Institute of Cytology RAS, Saint-Petersburg, Tihkoretsky ave, 4, 194064, Russia. nick.a.barlev@gmail.com.

Current Gene Therapy
|August 19, 2015
PubMed

Insights

Gene therapy, particularly using novel genome editing tools like CRISPR, offers a powerful strategy for treating genetic diseases, with over 60% of trials targeting cancer. These advanced molecular tools enhance precision and effectiveness in gene therapy applications.

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Gene therapy is a promising approach for treating genetic diseases, with a significant focus on cancer treatment.
  • Over 60% of the approximately 2,000 gene therapy clinical trials have targeted cancer.
  • Advances in molecular biology techniques are crucial for developing effective gene therapy strategies.

Purpose of the Study:

  • To review the current status of novel genome editing tools.
  • To discuss the suitability and perspectives of these tools in cancer gene therapy.
  • To highlight the impact of programmable nucleases and artificial transcription effectors.

Main Methods:

  • Review of recent advancements in genome editing technologies.
  • Analysis of programmable nucleases (ZFNs, TALENs, CRISPR) and their applications.
  • Discussion of DNA transposons as non-viral delivery systems.
  • Exploration of TALE- and CRISPR-based artificial transcription effectors for gene regulation.

Main Results:

  • Programmable nucleases (ZFNs, TALENs, CRISPR) offer high precision and efficacy in targeting specific genomic loci.
  • DNA transposons provide a promising alternative to viral vectors for gene delivery.
  • Artificial transcription effectors enable precise regulation of gene expression.
  • These tools have broad applicability in various gene therapy strategies.

Conclusions:

  • Novel genome editing tools represent a significant advancement in gene therapy.
  • Their precision and versatility hold great promise for the future of cancer gene therapy.
  • Continued development of these molecular tools will drive innovation in treating genetic diseases.

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