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This article was originally published by our sister site, Freethink.
For the first time, researchers appear to have effectively treated a genetic disease by injecting CRISPR therapy directly into the blood of patients, overcoming one of the biggest obstacles to curing disease through gene-editing technology.
The therapy appears to be surprisingly effective, altering almost every cell in the liver to stop a disease causing mutation.
The challenge: CRISPR gives us the ability to correct for genetic mutations, and since these mutations are responsible for more than 6,000 human diseases, the technology has the potential to dramatically improve human health.
One way to use CRISPR to treat disease is to remove affected cells from a patient, remove the mutation in the lab, and place the cells back in the body to replicate. This is how a team functionally cured people with sickle cell disease. , editing and then infusing bone marrow cells.
Bone marrow is a special case, however, and many mutations cause disease in organs that are more difficult to repair.
Another option is to insert the CRISPR system itself into the body so that it can make changes directly in affected organs (this has only been attempted once, in an ongoing study in which people received CRISPR therapy injected into their eyes to treat a rare vision disorder).
Injecting CRISPR therapy directly into the bloodstream, however, has been a problem, as the therapy has to find the right cells to modify. An inherited mutation will be in the DNA of every cell in your body, but if it only causes liver disease, you don’t want your therapy to be used in the pancreas or kidneys.
A new CRISPR therapy: Today, researchers at Intellia Therapeutics and Regeneron Pharmaceuticals have demonstrated for the first time that CRISPR therapy delivered into the bloodstream can travel to desired tissues to effect changes.
We can overcome one of the biggest challenges in the clinical application of CRISPR.
—JENNIFER DOUDNA
“This is an important milestone for patients,” Jennifer Doudna, co-developer of CRISPR, who was not on the trial told NPR.
“Although this is preliminary data, it shows us that we can overcome one of the biggest challenges in the clinical application of CRISPR so far, which is being able to deliver it systemically and ‘bring it to the right place,’ she continued.
What did they do: In a Phase 1 clinical trial, researchers at Intellia injected a CRISPR therapy called NTLA-2001 into the blood of six people with a rare and life-threatening genetic condition called transthyretin amyloidosis.
The liver of people with transthyretin amyloidosis produces a destructive protein, and CRISPR therapy has been designed to target the gene that makes the protein and stop its production. After a single injection of NTLA-2001, the three patients who received the higher dose saw their protein levels drop from 80 to 96%.
A better option: CRISPR therapy has produced only mild side effects and lowered protein levels, but we don’t yet know if the effect will be permanent. It will also take a few months before you know if treatment can relieve the symptoms of transthyretin amyloidosis.
It’s a wonderful day for the future of gene editing as a medicine.
– Fiodor URNOV
If all goes as hoped, NTLA-2001 may one day offer a better treatment option for transthyretin amyloidosis than a currently approved drug, patisiran, which only reduces toxic protein levels by 81% and should be injected regularly.
Looking forward: Even more exciting than the potential impact of NTLA-2001 on transthyretin amyloidosis, however, is knowing that we could use CRISPR injections to treat other genetic disorders that are difficult to target directly, such as heart disease or cerebral.
“It’s a wonderful day for the future of gene editing as a drug,” Fyodor Urnov, professor of genetics at UC Berkeley, who was not involved in the trial told NPR. “As a species, we are watching this remarkable new spectacle called: Our Gene Altered Future.”
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