Gene therapy, the modification of genetic information in living cells to address a mutated gene, has the ability to dramatically change the way diseases are treated, or even cured. First conceptualized in the early 1970s, the seminal paper on the subject was published in Science by Theodore Friedmann and Richard Roblin, suggesting the great potential for gene therapy techniques in human genetic disease.
The researchers also stressed that development should not proceed until and unless clearly defined ethical and scientific guidelines were outlined.1 The field suffered setbacks that made advancements challenging, including a patient death due to a severe immune reaction, and the development of leukemia in infants.2,3
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