Understanding AAV gene therapy

Genes are the blueprints of the human body. They instruct the body’s cells how to make proteins critical to their function. Many familiar and not-so-familiar diseases and disorders are caused by the faulty expression of proteins from corrupted or missing genes or by environmental modifications of genes or proteins.

Gene therapy uses modified viruses or other technologies to deliver therapeutic genes to cells or tissues to address genetic diseases at their source. One of the most exciting advances in modern medicine has been the discovery of how the adeno-associated virus (AAV) can be used as an effective delivery system for therapeutic genetic material into living tissue. AAV gene therapy has broad therapeutic implications for a vast array of diseases.

There are important benefits to using AAV as the primary means for delivering gene therapeutics:

  • AAV is a virus that is not known to result in human disease.
  • AAV cannot make more of itself without outside help, so it will not replicate in the body like normal viruses do. This lets scientists precisely control how much AAV will be given.

For these reasons, AAV has become the dominant form of gene therapy for genetic diseases. AAV is driving today’s therapeutic discoveries and is used in the only two FDA-approved gene therapies currently available.


How does AAV work?

Simply put, AAV is transformed from a naturally occurring virus into a delivery mechanism for gene therapy. The viral DNA is replaced with new DNA, and it becomes a precisely coded vector and is no longer considered a virus, as most of the viral components have been replaced. The AAV vector is then used to deliver normal copies of genes to the right tissues or organs in the body, but it now delivers the therapy that has been engineered into it.

Today, AAV technology has advanced to target a wide range of tissues and cells for the treatment of many genetic diseases. With some diseases, it is possible to put two copies of a gene into a vector, not just one. These vectors are called self-complementary AAV, or scAAV. Our co-founder, Dr. Jude Samulski, helped discover and refine this technology, and it is used in many therapies across the industry today. 

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