Journey from Jumping Genes to Gene Therapy

Katharine A Whartenby1

  • 1Departments of Neurology and Oncology, Johns Hopkins University School of Medicine, Baltimore, MD.

Insights

Gene therapy, a revolutionary cancer treatment, involves modifying human genetic makeup. This review explores the history, methods, and challenges of gene transfer in cancer therapy.

Area of Science:

  • Oncology
  • Genetics
  • Molecular Biology

Background:

  • Gene therapy for cancer is an evolving field with a long history rooted in genetic modification studies.
  • The concept of manipulating genetic material has evolved over centuries, from observing heredity to advanced human gene alteration.
  • Gene transfer has undergone significant development, marked by early discoveries and clinical trials for various diseases.

Purpose of the Study:

  • To provide a historical overview of gene transfer biology.
  • To explain the application of gene transfer to genetic diseases and early cancer therapies.
  • To review different cell modification methods, underlying theories, and encountered limitations.

Main Methods:

  • Historical review of gene transfer research and applications.
  • Analysis of early gene therapy clinical trials in oncology.
  • Examination of various gene modification techniques and their theoretical basis.

Main Results:

  • Gene transfer has a complex history, progressing from basic research to clinical applications.
  • Early gene therapy approaches for cancer involved various methods with associated challenges.
  • Understanding the historical context is crucial for advancing current gene therapy strategies.

Conclusions:

  • Gene therapy for cancer represents a culmination of extensive research in genetic modification.
  • The review highlights the historical trajectory, methodologies, and inherent limitations of gene transfer in cancer treatment.
  • Continued exploration of gene transfer biology and techniques is essential for future therapeutic advancements.

Related Concept Videos

Gene Therapy00:59

Gene Therapy

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...
28.0K
Gene Therapy00:59

Gene Therapy

4.7K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
1.3K
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
17.6K
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
81.1K
Exon Recombination02:32

Exon Recombination

The evolution of new genes is critical for speciation. Exon recombination, also known as exon shuffling or domain shuffling, is an important means of new gene formation. It is observed across vertebrates, invertebrates, and in some plants such as potatoes and sunflowers. During exon recombination, exons from the same or different genes recombine and produce new exon-intron combinations, which might evolve into new genes. 
Exon shuffling follows “splice frame rules.” Each exon...
4.3K