A genetic condition could potentially have a genetic treatment. This logical thinking has led to more than 50 years of gene therapy research. Today, gene therapy is being evaluated in multiple clinical trials to determine benefits and risks for a range of genetic conditions, including hemophilia A and B.
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. In the lab, functional genetic material is introduced into the cells and are then delivered back into the patient’s body (i.e. CAR-T therapy).
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.
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 (i.e. vector-based delivery). 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. This means the mutated gene would still be there and can still be passed on to a person’s offspring.
Here at HemDifferently, we’re interested in exploring the science behind gene transfer therapy.
Gene therapy has been explored as a potential treatment approach for more than 50 years.
More than 2500 gene therapy studies have been completed, are ongoing, or have been initiated worldwide.
To explore current research in gene therapy, visit ClinicalTrials.gov for more information.
Concept of gene therapy considered as a form of treatment in the journal Science
Dr. Gordon Vehar publishes a paper reporting successful factor VIII cloning
First gene therapy trial in humans
Lessons learned regarding risks related to potential for severe immune response in early gene therapy trial with non-adeno associated virus(non-AAV) vector
The Human Genome Project is completed
China approves the first gene therapy for the treatment of head and neck cancers
First gene therapy trial in hemophilia B using AAV vector technology begins
First gene therapy trial in hemophilia A using AAV vector technology begins
FDA issues guidance for hemophilia gene therapy research in the U.S.
Additional gene therapy technologies and applications are being researched in hemophilia
Mutations can come in multiple forms and are responsible for most genetic conditions, including hemophilia A or B and cystic fibrosis. Play our simple game, Mutation Pop, to identify four of the most common forms of genetic mutations that can lead to genetic conditions:
In this game you will have to quickly spot the mutated genes and pop as many as possible while allowing the functional genes to pass through. Find out if you have what it takes to earn the highest score!
Many gene therapies are in ongoing clinical trials for a range of conditions to determine the potential risks and benefits.
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