They added regulatory elements called promoters and enhancers to direct the gene’s activity. Scientists developed better viral vectors to deliver genetic therapies. In the early 2010s, gene therapy experienced a renaissance. In his case, the viral vector caused a fatal immune response. saw another early setback: the 1999 death of 18-year-old Jesse Gelsinger, after receiving gene therapy for a rare metabolic disorder. They reported the first cures in 2000, but within several years, five of the 20 treated children developed cancer. The viral vector that delivered the gene to their T cells had also activated an oncogene, triggering leukemia. Now in her 30s, de Silva is active in the rare disease community.Įuropean researchers in the 1990s focused on SCID-X1, another form of SCID linked to the X chromosome. Their success spurred more trials in the 1990s for the same form of SCID. The first paper, in 1984, showing that a virus could insert genes into cells.ĭoctors decided to deliver a healthy ADA gene into her blood cells, using a disabled virus that cannot spread in the body. Injections of a synthetic ADA enzyme helped, but only temporarily. Without ADA, her T cells died off, leaving her unable to fight infections. She was born with a severe combined immunodeficiency (SCID) due to lack of the enzyme adenosine deaminase (ADA). In 1990, 4-year-old Ashanthi de Silva became the first gene therapy success story. Gene therapy 1.0: First introduction of corrected genes Boston Children’s uses in vivo gene therapy for several disorders, including hemophilia and ornithine transcarbamylase deficiency.Īfter a rocky start, gene therapy is on fire, drawing keen interest from the biopharmaceutical industry. In vivo gene therapy involves direct IV infusion of the vector into the bloodstream or injection into a target organ like the eye. Boston Children’s is using this method for such disorders as sickle cell disease, adrenoleukodystrophy, chronic granulomatous disease and others. Ex vivo gene therapy removes cells from the patient, introduces new genetic material, packaged in a delivery vehicle called a vector, then returns the cells to the patient. Gene therapy falls into two main categories. In the past decade, gene therapy has become a reality for multiple diseases, especially those caused by mutations in a single gene. In 2003, the Human Genome Project wrapped up, giving us a complete blueprint of our DNA. In the early 1980s, David Williams, MD, and David Nathan, MD, at Boston Children’s Hospital published the first paper showing one could use a virus to insert genes into blood-forming stem cells. (Image: Adobe Stock Illustration: Sebastian Stankiewicz, Boston Children's)Īs early as the 1960s, scientists speculated that DNA sequences could be introduced into patients’ cells to cure genetic disorders. Despite its early checkered history, gene therapy is now becoming a standard of care.
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