Originally published July 20 2005
Scientists developing cell treatment for genetic disease
by Mike Adams, the Health Ranger, NaturalNews Editor
Scientists say they are getting closer to developing a technique that would allow them to treat several genetic disorders by injecting "zinc fingers" into cells, which would bind to miscoded strands of DNA and repair the defective genes.
A method for fixing miscoded DNA by injecting foreign genes into cells won headlines three years ago when doctors in France and Britain announced a handful of successful cures related to X-linked severe combined immunodeficiency disease, or SCID, also known as "bubble boy" disease.
In a paper published earlier this month, scientists at California biotechnology company Sangamo BioSciences showed that zinc fingers can be used to erase targeted portions of DNA without risk of harmful side effects.
"This doesn't just deliver a foreign gene into the cell," said Nobel Prize winner and CalTech President David Baltimore, who with a Sangamo paper co-author Mathew Porteus proposed this method to cure genetic diseases.
While such a therapy has been theorized for years by Baltimore and others, Sangamo scientists are the first to show test-tube results with human cells.
In a paper published June 2, Sangamo researchers showed how they were able to correct the defective gene in 18 percent of the T-cells extracted from the body of an X-linked SCID patient.
If successful in trials, Sangamo's technology would be the first successful gene therapy, three decades after the concept of curing diseases by tinkering with the genome was first proposed.
One trial that did succeed, but then ended in tragedy, was a 2002 French X-linked SCID trial that used retroviruses to deliver a new gene into the patients.
The zinc fingers weren't specific enough and they created so many double-stranded breaks in the DNA that a lot of the cells chose to commit suicide rather than try to repair all the breaks.
"If they can figure out how to optimize their zinc fingers for any spot on the genome, this could target any gene you want it to," said Wolfe.
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