Los Angeles Times: In the third gene-therapy success of recent weeks, French researchers have arrested the progression of the rare and fatal degenerative disorder adrenoleukodystrophy, which was at the heart of the popular movie "Lorenzo's Oil." The disease has stabilized in two boys who were 7 years old when the therapy was performed two years ago, the team reported today in the journal Science.
"This is a disease that never, ever stabilizes" on its own, said Dr. Katherine A. High of the Children's Hospital of Philadelphia, who was not involved in the research. "The fact that they were able to achieve that means they are getting a therapeutic effect."
This is the fifth disease for which gene therapy has been shown to be beneficial, said Dr. Theodore Friedmann of UC San Diego, who was also not involved. "That's a major achievement for a field that has been in the clinic for only 18 or 19 years. . . . This is a new form of medicine and deserves to be seen as such."
Indeed. Read the whole thing.
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