Gene Therapy
What are genes?1-4
Every cell in the human body contains chromosomes made up of genes. Genes consist of DNA, which provides instructions to cells on how to make proteins, which are essential building-blocks for our bodies. The human body contains thousands of genes. Boys with Duchenne have a change in the gene (known as a mutation) that makes dystrophin. Dystrophin is a protein that helps keep muscles working properly.
What is gene therapy? How does it differ from other treatment options for Duchenne?
Gene therapy is a treatment that involves the introduction, removal, or change in genetic material in a person’s cells. There are various approaches to gene therapy:
The insertion, replacement, or deletion of DNA within a gene.2-4
Changing the expression of RNA (a DNA-like molecule that helps the cell create proteins from genes) to block the production of dysfunctional protein from a mutated (altered) gene.
What is NAV® Technology?
REGENXBIO’s investigational gene therapies are designed to deliver working genes to cells using adeno-associated virus (AAV) vectors as delivery “vehicles.” These gene therapies use well-studied vectors from the NAV® Technology Platform, our proprietary gene delivery platform. Thousands of patients have received AAV gene therapies derived from the NAV Technology Platform.
1. Gene therapy. Mayo Clinic; 2021. https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619. Accessed December 22, 2021. |
2. How gene therapy can cure or treat diseases. U.S. Food & Drug Administration (FDA); 2022. https://www.fda.gov/ForConsumers/ConsumerUpdates/ucm589197.htm. Accessed January 15, 2025. | 3. What Is Gene Therapy? U.S. Food & Drug Administration (FDA); 2018. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy#:~:text=Gene%20therapy%20is%20a%20technique,that%20is%20not%20functioning%20properly. Accessed January 15, 2025. | 4. Khan S, Ullah MW, Siddique R, et al. Role of recombinant DNA technology to improve life. Int J Genomics. 2016;2016:2405954. doi: 10.1155/2016/2405954.
