CRISPR gene editing is good at repairing disease mutations in cultured cells in the laboratory.
Recently, researchers from the University of London in the United Kingdom injected CRISPR drugs into the blood of patients with a rare genetic disease (transthyretin amyloidosis) for the first time, and found that the livers of three of them almost stopped producing toxic proteins
Although it is not yet certain whether CRISPR treatment will alleviate the symptoms of the disease, the preliminary data of the study makes people excited about the effects of this one-time treatment
The principle of CRISPR clinical trials to treat transthyretin amyloidosis is to inactivate a mutant gene that causes human liver cells to produce a misfolded protein called transthyretin (TTR).
For most diseases, CRISPR therapy means injecting CRISPR components or genetic instructions into the blood in some way, and then targeting the treatment to an organ or tissue.
In this study, the subjects were four men and two women with transthyretin amyloidosis, aged 46 to 64 years old, and they were all injected with lipid particles containing two different types of RNA: one encoding the Cas protein This CRISPR component can cut DNA, and there is a guide RNA that can direct it to the TTR gene
According to Julian Gillmore, the head of the trial and University of London, after 28 days, the TTR levels of the three patients who received the higher dose of the two-dose treatment dropped by 80% to 96%, which was comparable to the average level of the patisiran drug treatment group (the TTR level dropped) About 81%) is the same or better
David Adams, a neurologist at the University of Saclay in Paris, led the trial of the patisiran drug.
It may take several months for patients receiving CRISPR treatment to see relief of symptoms, but according to reports, the therapy has few short-term side effects
Kenneth Chien, a cardiovascular researcher at Karolinska Institute in Sweden, said that lipid-encapsulated mRNA may be safer than using viruses to deliver genetic instructions that encode editing proteins and guide RNA into cells
University of California, Berkeley, Jennifer Doudna, said the new work is "a critical first step in the ability to inactivate, repair, or replace disease-causing genes in any part of the body
Related paper information: https://doi.
https://doi.
org/10.
1056/NEJMoa2107454
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