In what may be the first successful gene editing inside the body, two patients with a rare genetic disorder now have a corrective gene at very low levels, researchers say.
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Even though those low levels may not result in successful treatment of the two men, this is a significant advance in efforts to alter DNA in adults to treat diseases caused by defective genes, the Associated Press reported.
The preliminary results were presented Thursday at a conference in Orlando, Fla.
"This is a first step," said Dr. Joseph Muenzer of the University of North Carolina at Chapel Hill, who helped test the treatment and presented the results. "It's just not potent enough."
He consulted for the gene therapy's California-based maker, Sangamo Therapeutics. Efforts to develop a stronger version of the treatment are underway, the AP reported.
Sangamo's research involves men with Hunter or Hurler syndrome, who are missing a gene that makes an enzyme to break down certain sugar compounds. Without this gene, sugars accumulate and damage organs, and patients often die by their teens.
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