Major advance in gene therapy

Potential for editing genes directly in the body

Here is a brief video from the regulatory body in the US, the FDA, on the basics of gene therapy “Gene Therapy Inside Out”.

And if you want to learn more.

Up until now CRISPR-Cas9 gene editing technology has been an incredibly important tool for editing genes in the laboratory or experimental setting. When thinking about human application with this technology, cells needed to be taken from the body and the editing done in the laboratory… but that might be about to change!

from an article in Nature magazine:

Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease.

https://upload.wikimedia.org/wikipedia/commons/8/80/201412_CRISPRCAS9pink.png
CRISPR gene editing*

The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver — are infused into the bloodstream. In the trial, six people with a rare and fatal condition called transthyretin amyloidosis received a single treatment with the gene-editing therapy. All experienced a drop in the level of a misshapen protein associated with the disease. Those who received the higher of two doses tested saw levels of the protein, called TTR, decline by an average of 87%.

Previous results from CRISPR–Cas9 clinical trials have suggested that the technique can be used in cells that have been removed from the body. The cells are edited and then reinfused back into study participants. But to be able to edit genes directly in the body would open the door to treating a wider range of diseases.

“It’s an important moment for the field,” says Daniel Anderson, a biomedical engineer at the Massachusetts Institute of Technology in Cambridge. “It’s a whole new era of medicine.”

Researchers will anxiously await data from more participants, says Anderson, and will want to know how all the participants fare over a longer period of time. But the current results are enough to fuel hope that CRISPR–Cas9 will one day be able to help treat other conditions, too.

Techniques for delivering CRISPR–Cas9 components to various parts of the body are advancing rapidly, says Anderson. “The list keeps growing,” he says. “I’m optimistic that we’re going to see much broader application of genome editing.”

Read the entire article 

SOURCE:  https://www.nature.com/articles/d41586-021-01776-4  doi: https://doi.org/10.1038/d41586-021-01776-4

Landmark CRISPR trial shows promise against deadly disease

Heidi Ledford. NEWS 29 June 2021

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