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
Inside a cell
Simply put, gene therapy is being researched in clinical studies as a novel method that attempts to useto treat or prevent disease.
METHODS OF GENE THERAPY BEING EXPLORED
Gene transfer therapy
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.
Ex vivo gene therapy
- With the method of gene therapy called gene transfer, a functional gene is inserted into a 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 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 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 . 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. Gene therapy is currently being researched in many clinical trials for various genetic disorders, includingA and B, and has not been determined to be safe or effective.
To explore future, current and past research in gene therapy, visit ClinicalTrials.gov
GENE THERAPY TIMELINE
Gene therapy is in ongoing clinical trial research to determine the potential risks and benefits of treatment.