CRISPR used to treat rare genetic disease for the first time

In March of last year Doctors in Oregon are using CRISPR inside a person’s body for the first time. This time, another team of doctors in England. Once again, the revolutionary gene-editing tool was used in viv. This time for an experiment that attempts to treat people with a rare genetic disorder that causes a buildup of harmful proteins in their organs.

NIH

science The magazine reports on a new trial led by University College London Professor of Medicine Julian Gillmore and her colleagues conducted at the Royal Free Hospital in the British capital. It seeks to treat six patients for a rare genetic disease called hereditary transthyretin amyloidosis (or TTR).

To treat the six patients, Gillmore’s team used a combination of CRISPR and mRNA technologies, essentially delivering a “frequently clustered short palindromic duplication”, a collection of DNA sequences found. in the bacterial genome through the same type of fat particles or fat bubbles. which transmits the genetic code for the coronavirus-infected spike protein – uses 19 vaccines

UC Berkeley/Roxanne Makasdjian et al.

Doctors use DNA sequences within fat bubbles to target mutated genes within a patient’s liver cells. A malfunctioning gene creates an “abnormal” form of the transthyretin protein. which is built on nerves and heart leading to pain, numbness and heart disease

Transmission of the Cas protein genetic code, a DNA truncated CRISPR sequence in the image above—through a lipid bubble. Doctors were able to remove malfunctioning genes of liver cells. and most notably the reduction in volume. transthyretin The participants’ bodies were significantly reduced, up to 96% in one case, and on average more than 50%.

“This is good news for patients with this condition,” Gillmore said in a news release. “If this trial continues to be successful, The treatment may allow patients diagnosed in the early stages of the disease to lead a normal life without the need for continued treatment,” the professor added.

for the disadvantage doctor specific report However, it seems that many doctors and scientists not involved in the trial were encouraged by the results. Jennifer Doudna from the University of California Berkeley, who won last year’s Nobel Prize for CRISPR discovery, for example, told science that this work is “An important first step to inhibit, repair or replace any genes. that causes disease” anywhere in the body, which, frankly speaking, seems possible. but still far away

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