View Full Version : Lorenzo's Oil

Ballet Mom
11-05-2009, 01:25 PM
One home run for gene-therapy.

"ALD is the condition described in “Lorenzo’s Oil,” the 1992 movie starring Nick Nolte and Susan Sarandon and based on a family’s real-life search for a cure for their son. "


Gene Therapy Advance Saves Two Boys From Rare Brain Disease

By Rob Waters

Nov. 5 (Bloomberg) -- Two seven-year-old boys with a fatal brain disease who couldn’t get bone marrow transplants were saved by scientists whose gene therapy technique may let doctors treat other incurable disorders.

Doctors in Paris delivered the gene into the boys’ bodies using HIV, the virus that causes AIDS. The virus, stripped of genetic material that makes it toxic, integrates permanently into the DNA of cells it enters, scientists said. That means the modified gene remains in the blood-forming stem cells for the life of the patient, according to a report in the journal Science.

Two years after the experimental treatment, the neural damage has been halted or reversed and the two boys attend school and lead normal lives, said Nathalie Cartier, the study’s lead author. The treatment was cited as an example of a “comeback for gene therapy,” after years of setbacks, in an editorial in the journal.

“Their disease is completely stabilized, they are fine and there’s no reason this should change,” Cartier, research director at the French National Institute for Health and Medical Research in Paris, said in a telephone interview yesterday. If more studies confirm the results, she said, “We think gene therapy could become a first-line treatment. It’s definitely a strategy that could be applied to other conditions.”

Today’s report “represents a long-sought rewarding achievement in the field of gene therapy,” wrote Luigi Naldini, a researcher in Milan, Italy, in the editorial accompanying the study.

11-05-2009, 07:37 PM
That's great news.

Gene therapy is a simple concept. Let's say the part of your brain responsible for producing Melatonin isn't making any. Doctors will inject a virus into your body that contains a gene that turns that part of the brain on again or maybe programs a different part of your brain to produce Melatonin. Someday this basic idea may lead to cures for almost everything.

Today health care costs are rising fast but 30 or 40 years from now they may be headed in reverse because most chronic disease will be curable.

Ballet Mom
11-06-2009, 10:53 AM
Hi Dingo,

Yes, it is truly a very exciting time to be living in. It never ceases to amaze me that there is usually a good or useful side to very destructive things....the AIDS virus, of all things, being used for a good purpose. Stunning.