LA JOLLA (CNS) - Researchers have found a method of safely replacing defective genes with healthy ones through a process called "gene editing," the Salk Institute for Biological Studies announced Friday.
Researchers were able to introduce molecules with healthy DNA into cells, with the healthy DNA swapping places with defective DNA, sort of like the "find and replace" function on a word processor, according to the institute.
Scientists used pluripotent stem cells, which can be developed from a patient's own skin and grown in a laboratory, and adult stem cells.
"The ability to derive and grow human pluripotent stem cells in the laboratory has raised enormous expectations within the biomedical community due to their transplantational potential in clinical settings," said Izpisua Belmonte, a professor in the Gene Expression Laboratory and the study's leader.
"This, when combined with the development of efficient and safe gene editing technologies in human stem cells may greatly help the realization of these expectations."
Gene editing overcomes the limitations of gene therapy, which has only temporary effects, according to the institute.
"The process was remarkably efficient and we couldn't detect any undesired off-target effects such as genomic instability or epigenetic abnormalities," said Guang-Hui Liu, a post-doctoral researcher and one of the report's authors. "What's more, it allowed us to show that we can correct multiple mutations spanning large genomic regions."
The research focused on the lamin A gene, on which 400 different mutations have been reported and which is associated with a number of illnesses.
Gina Kirchweger of the Salk Institute said one of the best applications for the new method could be for adult muscular dystrophy patients.
She said a recent study at UC San Diego that showed the immune systems of laboratory mice rejected infusions of pluripotent stem cells did not apply to the Salk Institute findings, which are set to appear in the June 3 issue of the journal Cell Stem Cell.
