CRISPR gene-editing breakthrough opens door to treating broad array of diseases

&gt;Doctors infused billions of microscopic structures known as nanoparticles carrying genetic instructions for the CRISPR gene-editor into four patients in London and two in New Zealand. The nanoparticles were absorbed by their livers, where they unleashed armies of CRISPR gene-editors. The CRISPR editor honed in on the target gene in the liver and sliced it, disabling production of the destructive protein.<p>this is one of the coolest paragraphs I&#x27;ve read in a while. if it&#x27;s accurate, serves as a great reminder that we&#x27;re in the future as we speak

Reminds me of this guy that cured himself of lactose intolerance. <a href="https:&#x2F;&#x2F;www.popularmechanics.com&#x2F;science&#x2F;health&#x2F;a17804128&#x2F;scientist-painstakingly-documents-his-own-diy-gene-therapy&#x2F;" rel="nofollow">https:&#x2F;&#x2F;www.popularmechanics.com&#x2F;science&#x2F;health&#x2F;a17804128&#x2F;sc...</a>

Off topic: are there any central repositories tracking gene therapy progress for various genetic conditions, with a similar UX to Moderna’s mRNA pipeline tracker?<p><a href="https:&#x2F;&#x2F;www.modernatx.com&#x2F;pipeline" rel="nofollow">https:&#x2F;&#x2F;www.modernatx.com&#x2F;pipeline</a>

A lot of times you hear about these breakthroughs in medical papers or in mice, but this really feels different.<p>Like an actual breakthrough with an actual cure for a specific disease. It&#x27;s remarkable.<p>Here&#x27;s to more cures.<p>God bless.

Crazy genetics ideas I would like explored<p>A modified tree that drops nuts of (nearly) pure graphite or some other form of nondecomposable carbon which we can easily harvest and sequester.<p>Photosynthesis in humans to supplement our energy consumption. Shoutout to Knights of Sidonia.<p>I understand the ethical issues surrounding the second one.

So this is editing liver cells, which I read somewhere is the easiest to do, since the liver absorbs foreign elements from the bloodstream.<p>The success of this should mean that many genetic liver conditions can soon be treated this way.<p>For other organs, I expect a longer wait.

The fact that we can apply these sort of therapies to an adult human who consists of millions upon millions of cells blows my mind. Having the ability to fix our &quot;buggy&quot; genetic code while we are alive is amazing.

I feel like I missed something, and now everywhere I look we have headlines like this.<p>What&#x27;s wrong with &quot;CRISPR gene editing shown to cure Transthyretin amyloidosis in patient study&quot;<p>Maybe I&#x27;m now one of the old people who doesn&#x27;t understand new usage of language. But I understand that usage dictates grammar rules, but I feel I missed something.

Please do familial hypercholesterolemia soon! I know it would be harder but getting started on targeting specific mutations could be fruitful.<p><a href="https:&#x2F;&#x2F;www.ncbi.nlm.nih.gov&#x2F;pmc&#x2F;articles&#x2F;PMC3681164&#x2F;" rel="nofollow">https:&#x2F;&#x2F;www.ncbi.nlm.nih.gov&#x2F;pmc&#x2F;articles&#x2F;PMC3681164&#x2F;</a>

Wikipedia says that they first used viruses as delivery agents; now it&#x27;s nanoparticles, aparently to avoid destruction by the immune system. Also interesting that CRISPR gene editing was also the subject of a patent war. Amazing stuff... (also it took them less than a year to adopt the nanoparticle trick to CRISPR gene editing. Things are moving fast)<p><a href="https:&#x2F;&#x2F;en.wikipedia.org&#x2F;wiki&#x2F;CRISPR_gene_editing" rel="nofollow">https:&#x2F;&#x2F;en.wikipedia.org&#x2F;wiki&#x2F;CRISPR_gene_editing</a><p><a href="https:&#x2F;&#x2F;news.harvard.edu&#x2F;gazette&#x2F;story&#x2F;2020&#x2F;11&#x2F;coated-nanoparticles-survive-immune-system-and-deliver-drugs&#x2F;" rel="nofollow">https:&#x2F;&#x2F;news.harvard.edu&#x2F;gazette&#x2F;story&#x2F;2020&#x2F;11&#x2F;coated-nanopa...</a>

It&#x27;s only a matter of time before we use this to cosmetically modify our bodies. I predict that at the very least we&#x27;ll use this technic to change our hair color and eye color. I will even say skin color too, with-in the next 50 years.<p>Most people won&#x27;t use it but there will be an active market , legal or illegal, for the modifications. The Sci-Fi future is here.

<i>We have tried every imaginable remedy. In some instances there has been brief recovery, followed always by a still worse relapse. Physicians who are familiar with alcoholism agree there is no such thing as making a normal drinker out of an alcoholic. Science may one day accomplish this, but it hasn&#x27;t done so yet.</i> -- Alcoholics Anonymous Chapter 3, More About Alcoholism<p>I may soon be able to drink again after having my genes edited, but I&#x27;m not sure I would do it

The period missing from the title makes this very hard to parse what is being said.

Wow...this is a really big deal. Congratulations to this team and to the patients who were treated. I hope the treatment continues to keep the disease at bay.

Original paper: <a href="https:&#x2F;&#x2F;www.nejm.org&#x2F;doi&#x2F;full&#x2F;10.1056&#x2F;NEJMoa2107454?query=featured_home" rel="nofollow">https:&#x2F;&#x2F;www.nejm.org&#x2F;doi&#x2F;full&#x2F;10.1056&#x2F;NEJMoa2107454?query=fe...</a><p>This is the first published IV-administration of a gene-editing CRISPR payload. Interesting convergence with the nanoparticles used in mRNA vaccines. Sickle-cell and immune reprogramming edits have been made on cells removed from the body. Josiah Zayner infamously injected himself with CRISPR at a conference years back, but (1) unpublished, AFAIK and (2) he had been drinking which likely attenuated the payload [and (3) he wanted bigger muscles, not to treat a disease]

Turning medical science from a technical problem into an ethical one would be a massive epoch boundary for humanity, I’d say. More so than the Internet.

This is just mind blowing.<p>What are some good investment opportunities if you think this is going to revolutionize the world in the next 30-50 years?

This is awesome but I&#x27;m curious why they tried it first on this super-rare disease instead of something more common?

I&#x27;m very hopeful that someday these techniques will cure diseases like Huntington&#x27;s

I must be out of the loop. Last I heard trials with CRISPR were making not only the desired genetic changes but also dozens of others at random points in the genome. Did they solve that problem?

Wow, it worked...This uses LNPs similar to the recent Moderna and BioNTech&#x2F;Pfizer vaccines, and N1m-pseudouridine mRNA, just like the mRNA vaccines. This is incredible.

Can anyone explain how this survives being in the blood stream and then how it actually gets into the nucleus of the cell?

Wonder how long it will be until this is used to enhance rather than just to cure.<p>Eg selective fast twitch &#x2F; slow twitch muscle fibres.

What are the limits of gene editing?

Could we hack our genes to need less oxygen and eat less?<p>That could be useful in the future.

Stupid question: If he were to have kids, would they inherit this fix?

Want to try chameleon color changing dna to pomerian dog

Not reading the entire article - I believe this is going the wrong way…

So the religious right decided that we do not own our bodies, god does. Just as software companies issue a EULA, all children born since 2032 are required to have their parents sign a EULA. Genetic modification is strictly prohibited and punishable by reduction of the government stipend that everyone receives in fedcoin every month. Underground genetic modification labs are run by organized crime but unfortunately those using these labs also get an unplanned genetic modification in addition to the one they want. They are made dependent on a drug that only those labs can provide, for a price. In addition, there are monthly vaccination programs for citizens to protect them against genetic terrorists, carriers of lethal protoviruses who are engineered to carry and disseminate Covid derivatives that target HLA SUBTYPES common in Northern European and South American genotypes.<p>Yes genetic modification is another wonderful technology that humans will weaponize….like atomic energy, like the internet…

Cool, we can only show stories where it worked