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.
West Nile virus made its first appearance of the summer in San Diego County this week when a batch of mosquitoes trapped in Santee tested positive for the viral infection.
The San Diego County Medical Examiner’s Office is asking for the public’s help to identify a woman, possibly in her late teens, killed by a hit-and-run driver as she walked along state Route 94 earlier this month.
Major traffic is expected to start Wednesday evening as Comic-Con kicks off with preview night in Downtown San Diego. MTS officials are encouraging attendees to use public transportation and got into the SDCC spirit with an homage to the "Stranger Things."
A person was struck and killed Wednesday by an SUV on Interstate 805 in the Lincoln Park area, necessitating the closure of multiple freeway lanes during the morning commute.
A 28-year-old man died Wednesday after he was found shot in a car in San Diego's University Heights neighborhood, police said.
Calling all aspiring entomologists! You're invited to San Diego Botanic Garden’s Insect Festival this weekend!
Comic-Con International, again expected to draw a wide array of fans of the popular arts, will conduct its annual preview night at the San Diego Convention Center Wednesday night.
A 25-year-old man suffered a fractured femur today when he was shot in the right leg by a man in San Diego's Chollas View neighborhood, police said.
Lanes were closed Wednesday on Mission Gorge Road between Fanita Drive and Carlton Hills Boulevard because of a water main break, officials said.