Gene therapy is a technique that works to modify a person’s gene(s) to treat or cure a disease. This technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells to help fight a disease instead of using drugs or surgery. For gene therapy to be successful a safe delivery of genetic cargo is key. There are multiple viruses that can be used to deliver genes to cells and each virus has its advantages and disadvantages.
Gene therapy studies using Adeno-Associated Virus (AAV) vector have shown significant progress in human gene therapy and AAVs have emerged as the predominant vectors for delivering genes of interest to target tissues with improved specificity, efficiency, and safety.
AAV vectors are bioengineered tools that use a non-enveloped virus to transport modified genetic material safely into tissues and cells impacted by otherwise difficult-to-treat conditions. These vectors deliver their genetic cargo into tissues, after which the modified genes will create new instructions for those tissues and help treat disease.