First Human To had His Genes Edited By Sangamo Therapeutics
So a week ago I stumbled upon an article by Futurism describing the first patient being CURED from Hunter's Syndrome, a genetic disease which prevents the sufferer to produce enzymes that breakdown complex carbohydrates, resulting in their build-up in the body.
Hunter's Syndrome is caused by a recessive gene on X chromosome, meaning that if you have one properly functional gene, you're not going to get sick
The disease has many complications, from heart problems, neurological problems such as seizures, agressiveness and cognitive development issues. And the average life expectancy of someone with Hunter's Syndrome is usually between 10-20 years, but mild and more well managed patients live much longer, E.G. 44 years for the recipient of the new treatment.
The build-up of complex carb molecules in connective tissues results in abnormalities in bones, joints and ligaments
The company (Sangamo Therapeutics) used a technique called ZFN (Zinc finger nucleases), which is essentially creating an enzyme that cuts the DNA at certain places, just to insert a few base pairs, which will act as a new gene, giving the cell new functions.
The Company Behind The First Humans Trial
Another popular technique being researched (currentrly still on animals) is CRISPR, which uses enzymes found in bacteria (which are a part of it's defense mechanism against viruses) that is capable of cutting and removing and\or adding genes.
I've found an article explaining the use of CRISPR to edit RNA (what actually reads DNA and makes the proteins), which is much safer, because the changes to it AREN'T PERMANENT, the researches have succesfully altered the function of RNA of defective genes, as if the genes that the RNA read were healthy, specifically genes that produced anemia and diabetes if untreated.
The team behind the CRISPR trial used Cas13b, which allowed to edit RNA, instead of using the RNA to edit the DNA itself
So, what do you think should this technology be used for? Is curing diseases enough? Or enhancing human abilities is on the table?
I mean there was a study that found several genes that were highly correlated with musical ability.
So should people be allowed to have their music skills enhanced?
Let me know in the comments below, and follow me, if you'd like to see more articles like this one in your feed :)
Sources
Hunter's Syndrome Complications
Hunter's Syndrome Life Expectancy
General Information About CRISPR
That is most interesting article @imacryptorick, with the great hope in the new medicine methods, but also opening a great field for misuse… Worth following.
Thank you ^_^
very interesting and a good scientific topic to be read
Thanks :)
Wow.... Great topics.... But I have not any idea about this... I also want to know that... Can anyone help me please....??
A very good post..thank you..upvote.greets
I think this is a medical breakthrough, i maybe new but it is worth to try. The disease look horrible. Im gonna look this up and search more about it.