World's very first gene therapy recipient was a Sri Lankan young child (now in her twenties) in the US.Ashanthi de Silva.
An excerpt from an article: Anderson struggled until 1984, when MIT researcher Richard Mulligan devised a way to safely insert genes into cells using a retrovirus rather than a needle. At the time, no one knew what would happen to a person who had a foreign gene inserted — a complex process in which the patient's cells were "infected" by a virus that had been genetically modified to include human DNA. But Anderson believed profoundly in the power of gene therapy to cure inherited diseases and wanted to test that theory. Although experiments in animals seemed to show that the procedure was safe, regulators were wary, and in 1988, the NIH's Human Gene Therapy Subcommittee unanimously denied Anderson's proposal to begin human testing. Anderson demanded a hearing before the full committee, many of whom were nonscientists. The idea, Anderson explained after the fact, was to "change the playing field" through a "carefully planned emotional appeal."
"I was asked, ‘What's the rush in trying to get your protocol approved?'" Anderson said during the hearing. "A patient dies of cancer every minute in this country. Since we began this discussion 146 minutes ago, 146 patients have died of cancer."
Anderson's trial was approved 16 to 5, with the dissenting votes cast by the five molecular biologists on the committee who stressed the lack of good test results in animals. A few months later, in May 1989, he conducted the first human safety test for gene therapy, inserting a harmless marker gene into a 52-year-old man. A year later, Anderson took the next step: giving 4-year-old De Silva a transfusion of blood cells that had been genetically modified to include a functioning ADA gene, which helps the body counter infections. Although De Silva's immune disorder — caused by a mutation in the gene that produces ADA — could be partly controlled through an artificial supplement, PEG-ADA, Anderson hoped to create a permanent cure.
Now 21, De Silva is a senior in college. Although she continues to need PEG-ADA, her health has been fairly stable. Her mother, Van De Silva, believes that the injections of genetically enhanced blood administered by Anderson saved Ashanthi. "If not for him, I don't think my daughter would be alive," she says fervently. "He's a good man. A good man."