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A team of researchers found that DMD in laboratory mice had been corrected for more than a year after a single gene editing CRISPR treatment. They hope that their study will pave the way for the development of effective treatment for humans. ( Duke University )
Researchers at Duke University have used the CRISPR gene editing to correct the genetic disorder called Duchenne Muscular Dystrophy, or DMD, in mice.
The team found that the technology could safely fix DMD and remain stable for over a year. They hope that this study will lead to the future use of gene editing for human therapy.
The researchers published an article entitled "Long-term Evaluation of the AAV-CRISPR Genome Edition for Duchenne Muscular Dystrophy" in the journal Nature Medicine Monday, February 18th.
Gene Editing in Treatment of Muscular Dystrophy in Mice
DMD is a rare genetic disorder that occurs when body cells can not produce dystrophin, resulting in muscle weakness that worsens over time. Many patients diagnosed with the disease are wheelchair bound at the age of 10 and do not live beyond their 30s.
DMD does not exist at present, but several researchers, including Charles Gersbach, professor at Duke University, are exploring the possibility of using gene editing for the treatment of genetic diseases.
In their new research, the team led by Gersbach used the CRISPR / Cas9 tool to cut the exon of the mutation's dystrophin to allow the natural system of DNA to 39, bademble the remaining gene. The result is a shortened but functional dystrophin gene.
During the experiment, the researchers administered CRISPR treatment to adult and newborn mice. For one year, they measured the number of muscle cells successfully edited and monitored the genetic changes that occurred. They also took note of the immune system's response to the Cas9 CRISPR protein, which acts as a chisel that "cuts" into the genome.
In the first eight weeks, researchers found that functional dystrophin was restored in mice, resulting in increased muscle strength.
The team observed a response from Cas9, but said it did not result in the death of mice. It also did not interfere with the ability of the therapy to edit the dystrophin gene.
The researchers also observed that two-day-old mice receiving treatment had no immune response. In some cases, they found that the CRISPR gene editing was enhanced during the course of the year.
Towards the treatment of DMD
Gersbach and his team said the purpose of the study was to explore the long-term potential consequences of gene editing as a treatment for genetic diseases, particularly DMD.
"It is widely accepted that gene editing leads to permanent gene correction," he said. "However, it is important to explore the theoretical possibilities that could undermine the effects of gene editing, such as the loss of treated cells or an immune response."
Christopher Nelson, a postdoctoral fellow at the university, added that their findings suggest that the immune system's response to the Cas9 should be explored further, while researchers are preparing to test CRISPR on the same day. other animal models and possibly on the human.
Gersbach has been working on a potential DMD treatment with CRISPR since 2009. In 2016, he and his team published one of the first "successful uses of CRISPR to treat an animal model of [a] genetic disease."
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