An American start-up company successfully treated the first patients with Crispr gene editing therapy, which targets internal organs in the body.
Early trial data from Intellia Therapeutics co-founded by Nobel Prize winners Jennifer Dudna, Marking a breakthrough Crispr-based treatment, Indicating that scientists have overcome previous challenges that limited the use of this technique for editing in vitro or eye cells.
This Boston-based startup teamed up with biotechnology company Regeneron to treat transthyretin amyloidosis, a devastating disease in which the accumulation of problematic proteins can affect the heart and nerves of patients System, thereby shortening their life expectancy.
Intellia’s chief executive, John Leonard, stated that he was “very happy” to see the positive results. Beyond treatment A “small subset” of diseases based on Crispr-based treatments has been tested.
“The charm and prospect of Crispr is that you can change any gene anywhere in the genome, as long as you can. The last condition is the key,” he said. “This is the first time Crispr has been injected into a patient… the first time we can successfully target genes.”
Crispr—representing clusters of regularly spaced short palindrome repetitions—is a system used by bacteria to protect themselves from viruses. In 2012, Doudna and her French colleague Emmanuelle Charpentier discovered how Use it as a gene editing tool.
Since its listing in 2016, Intellia’s stock price has risen 233%. The company is one of three companies that hold the original patents for these discoveries.The others are Crispr therapy, Treating patients with sickle cell disease, and Editas Medicine, are experimenting with a type of genetic blindness.
Intellia is seeking to edit bone marrow to treat blood-based diseases without transplanting cells, including working with the Bill and Melinda Gates Foundation to treat patients with sickle cell disease in Africa.
In its first phase of trials, Crispr treatment was inserted into lipid nanoparticles, which were absorbed into the blood by the same tissue that absorbed the cholesterol globules and transported to the liver. There, the one-time treatment inactivated the TTR gene and reduced the problematic protein by 87% in the highest-dose patients. There were no serious side effects by the 28th day.
Julian Gillmore, a professor of medicine at University College London, is the lead investigator of the phase 1 trial. He has treated patients with this type of amyloidosis for 25 years, but he has nothing to do for 20 years. In the past five years, he has been able to use gene silencers-but these treatments do not seem to be effective and require regular infusions.
He said: “From my personal point of view, watching these patients’ conditions deteriorate for so many years, I know that these families have been completely destroyed by this disease over the years. It is incredible to see this revolution.
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