Vanderbilt University Medical Center investigators are reporting success with a novel gene therapy approach. Working with cells grown in the laboratory, the group is the first to repair a defective gene and demonstrate that the resulting protein product is functional, said Dr. Alfred L. George Jr., senior author of a study published Dec. 15 in the Journal of Clinical Investigation.
Although use of the approach in patients is still years in the future, the findings are an important step in showing that a particular method of gene repair is possible, said George, director of Vanderbilts division of Genetic Medicine.
"We have very solid evidence that we can repair messenger RNA (the copy of DNA that is used to manufacture proteins), and that the repair results in a protein that has normalized function," he said. "Thats a good sign and makes us optimistic about moving forward with this type of gene therapy strategy."
Leigh MacMillan | EurekAlert!
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