The Current Status of Gene Therapy for the Treatment of Cancer

Tafere Mulaw Belete1

  • 1Department of Pharmacology, College of Medicine and Health Sciences, University of Gondar, Gondar, Amhara Region, Ethiopia.

Insights

Gene therapy utilizes genome engineering to treat genetic and acquired diseases. Advances in gene delivery systems and genome editing technologies like CRISPR/Cas9 offer new therapeutic strategies for various disorders.

Area of Science:

  • Biotechnology
  • Genetics
  • Molecular Biology

Background:

  • Gene therapy has evolved from managing genetic diseases to addressing a wider range of acquired and inherited disorders.
  • Genome engineering technologies have significantly advanced gene therapy over the past three decades.
  • Over 3000 genes are linked to diseases, with approximately 2600 gene therapy trials underway.

Purpose of the Study:

  • To review the principles and mechanisms of genome-editing technologies.
  • To explain various viral and nonviral gene delivery systems.
  • To summarize approved gene therapy medicines for cancer treatment.

Main Methods:

  • Review of genome-editing tools including zinc finger nucleases, TALENs, meganucleases, and CRISPR/Cas9.
  • Explanation of viral vectors (adenoviral, AAV, HSV) and nonviral delivery methods (physical and chemical).
  • Detailed summary of gene therapy drugs for cancer, including indications and vectors.

Main Results:

  • Genome editing offers precise gene modification capabilities.
  • Diverse delivery systems are available for effective gene transfer.
  • Several gene therapies are approved for treating various cancers.

Conclusions:

  • Gene therapy presents a promising alternative for managing numerous diseases.
  • Safe vectors and advanced biotechnologies are crucial for future gene therapy applications.
  • Gene therapy holds significant potential for disease prevention and treatment.

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