About Gene Therapy

Gene therapy is an investigational approach with the goal of treating or possibly preventing disease. Scientists have been investigating and evolving gene therapy for more than 50 years. One goal of gene therapy research is to determine whether a new or functional gene can be used to restore the function of, or inactivate, a mutated gene. At Spark Therapeutics, we are using adeno-associated viral (AAV) vectors to advance research programs against strategically selected cell targets, for example, the retina, liver and central nervous system.


Gene therapy resources