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What is gene therapy?

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A genetic condition could potentially have a genetic solution, right?

This logical thinking gave rise to the first research in gene therapy more than 50 years ago. Now being evaluated in multiple clinical trials for a range of genetic conditions to determine the possible benefits and risks, gene therapy research has the potential to bring an entirely new option to people with specific genetic conditions and those who support and care for them.

GENE THERAPY SEEKS TO ADDRESS GENETIC MUTATIONS

Simply put, gene therapy is being researched in clinical studies as a novel method that attempts to use genes to treat or prevent disease.

METHODS OF GENE THERAPY BEING EXPLORED

A few different approaches to gene therapy are being explored. Gene therapy may involve attempting to repair or replace a mutated gene, disabling a mutated gene that is causing trouble or introducing a functional copy of the gene into the body to help the body produce the affected protein.

  • With the method of gene therapy called gene transfer, a functional gene is inserted into a cell with the intent that it will work in place of the mutated gene. Viral, chemical and physical methods are being explored for transferring genes. The transfer of the new gene occurs inside the body (in vivo) after systemic delivery, often using an IV infusion. The original genetic material in the chromosomes is intended to be left unchanged. Gene transfer is not designed to replace or edit the existing gene. Therefore, the mutated gene could still be passed on to future generations
  • In ex vivo gene therapy, a type of cell-based therapy, the process happens outside the body. First, affected cells are removed from the body via a biopsy. In the lab, functional genetic material is introduced into the cells, which are then delivered back into the patient’s body
  • In gene editing, the idea is to make changes to the original DNA. This technique makes it possible to repair the original DNA or add new DNA in a specific location. Zinc finger nucleases and CRISPR (clustered regularly interspaced short palindromic repeats) are methods of gene editing being researched

50+ YEARS OF RESEARCH

Gene therapy has been explored as a potential treatment approach for well over 50 years. In the past decade, the US Food And Drug Administration (FDA) and the European Medicines Agency (EMA) have approved gene therapies for genetic conditions including haemophilia A and B.

To explore future, current and past research in gene therapy, visit ClinicalTrials.gov

GENE THERAPY TIMELINE

1972

Concept of gene therapy considered as a form of treatment in the journal Science

1984

Dr. Gordon Vehar publishes a paper reporting successful factor VIII cloning

1990

First gene therapy trial in humans

1999

Lessons learned regarding risks related to potential for severe immune response in early gene therapy trial with non-adeno associated virus(non-AAV) vector

2003

The Human Genome Project is completed

2003

China approves the first gene therapy for the treatment of head and neck cancers

2005

First gene therapy trial in hemophilia B using AAV vector technology begins

2015

First gene therapy trial in hemophilia A using AAV vector technology begins

2017

the first gene therapy, for a genetic disease that causes blindness, is approved in the United States

FUTURE

additional gene therapies are being researched

ONGOING RESEARCH

Gene therapy is in ongoing clinical trial research to determine the potential risks and benefits of treatment.