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Source: NBC News

History of Cell & Gene Therapy

History of Cell & Gene Therapy

In 1989, a landmark study at the National Institutes of Health (NIH) provided the first direct evidence that human cells could be changed and returned to a patient without harm.1 This research shifted how physicians and medical researchers approached previously incurable diseases, such as aggressive forms of cancer, inherited errors of metabolism, and even brain diseases.

In the early years, cell and gene therapy researchers faced challenges, including one that led to the tragic death of Jesse Gelsinger following experimental gene therapy in 1999,2 which was followed two years later by a patient who developed a blood cancer after treatment.

Despite setbacks, NIH and the international scientific community prioritized funding for transformative research, which has resulted in significant scientific advances, including the development of cell and gene therapies.

Today, various cell and gene therapies are transforming medicine and changing lives. In fact, diseases currently have approved cell and gene therapy options, and more than 1,000 clinical trials, where various therapeutic options are being rigorously evaluated, are being conducted right now.


1 Rosenberg SA, Aebersold P, Cornetta K, et al. Gene transfer into humans – immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990; 323: 570-8.

2 Wilson JM. Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency. Molecular Genetics and Metabolism 96 (2009):151-7.

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