>Using chemical design and synthesis, the team brought together the Nobel-prize winning technology with therapeutic technology born in their own lab to overcome a critical limitation of CRISPR. Specifically, the groundbreaking work provides a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. The team developed a way to transform the Cas-9 protein into a spherical nucleic acid (SNA) and load it with critical components as required to access a broad range of tissue and cell types, as well as the intracellular compartments required for gene editing.

Great to see more techniques around crispr keep being developed.

So could this cure eye issues? When can my asthma be fixed?

Normally I'd raise an eyebrow and wonder if we never heard of this again or if it became an integral technology, but given the 'delivery, delivery, delivery' mantra we see in the area this could be more likely to be significant than most.

Excited to see what therapies come up over the next century.

!RemindMe 10 years

I will be messaging you in 10 years on 2032-10-14 18:27:25 UTC to remind you of this link

11 OTHERS CLICKED THIS LINK to send a PM to also be reminded and to reduce spam.

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Do anyone actually know someone who has had crispr-based therapy?

Can it cross the blood/brain barrier?

Therapies can ya

This could make it easier to design gene drives for humans. It would be the perfect birth control for unwanted populations after robot labor takes over the heavy lifting.

Genuine question out of curiousity: does this imply that muscular dystrophy could be treated better, and vice versa it could be used as a way to dope up for sportsman and body builders? I mean gene editting doesn't strike me as something that could be easily proven, or?

That's exactly what I was wondering. I've been hearing about Crispr for I think more than 10 years, but I am yet to hear about Crispr being actually used as a treatment. Why is that ?

There are only a small amount of super expensive custom drugs available... It's probably not going to be until the end of the decade until we start seeing it more widespread.

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GPT-3 summary:

The breakthrough discussed in this article is a new platform for gene editing that could inform the future application of a near-limitless library of CRISPR-based therapeutics. This breakthrough is significant because it provides a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. This breakthrough works by transforming the Cas-9 protein into a spherical nucleic acid (SNA) and loading it with critical components as required to access a broad range of tissue and cell types, as well as the intracellular compartments required for gene editing. The article discusses how this breakthrough could help gene editing technology infiltrate cells and overcome a critical limitation of CRISPR.

!RemindMe 10 years

In a decade we may be talking about the eradication of a lot of genetic disease

Lemme guess “the researchers say it will still be years before the technique is available to humans. They hope to start clinical trials within the next 5-7 years.”

I genuinely love this science. I love that it got posted. I read thousands of pages on it. And I’ve become so bored with news articles about it.

>!RemindMe 10 years

!RemindMe 2 years

CRISPR wast invented in 2012.

We need those 10 or so years for more research and development. Humans have had CRISPR used on them many times already, but it's still early days for human trials. Most of the good stuff with CRISPR probably won't start appearing until the late 2020s.

Big Pharma

They still haven’t figured out hair growth or penis enlargement - future you

Jk jk

To all of you who wonder it science never leaves the lab - medical takes longer than material science for example, but don't you all remember foldable displays being showcased at trade shows for like 4 years and we all thought "this never gonna go into co sumer product, it's vapor", and now we are at 4th gen consumer foldables and people aren't even surprised with those. Take Samsung flip and take it to a timeline 15 Years back - it looks and feels like a movie prop, no way it's real..

Medicine is the same we know it, in 1960, 1990 etc people were hearing about scientific concepts and those are now in the mass market from Lasik, to some Stem Cells therapies to HIV that used to be a death sentence similar to rare forms of cancer today, it not longer is....

Pace is a bit irritatingly slow compared to GPU progress and ability to simulate water in fps games (kidding) but it is also getting faster...

This is a good article that summarizes the state of CRISP research: [CRISPR Clinical Trials: A 2022 Update] (https://innovativegenomics.org/news/crispr-clinical-trials-2022/)

It’s probably just that medical technologies take a long time to be approved.

TLDR: drugs in a nutshell.

Its because we don't know what effects a drug will have until we actually inject it. You have to realize that our entire body is a diffusion prone liquid chamber of a billion moving parts but we need to deliver soluble drugs to a very specific target number of cogs. That's physically impossible btw.

So we end up having unknown side effects, and every drug has them. We just have to be persistent and select the ones that are effective at curing yet tolerable enough, but this process can take upwards to a decade in clinical trials.

In practice this means we have a decade long backlog of drugs that we think are promising but haven't had the time or resources to check. We'll get to testing CRISPR in maybe a few more years.

One more thing, as a scientist who has used CRISPR systems in cell cultures, its not that 'robust' yet. If I had to choose between successful CRISPR and fliiping a coin in a game of russian roulette, I'd still take the coin lol.

But... we are already testing CRISPR. I think the first human trials were 5 or 6 years ago.

I don't think you know about big pharma. They want these things by yesterday, because of money, you know? Always have but are heavily slowed down by the FDA's guidelines demanding all evidence in the world that this won't ever ever backfire in humans. This means more time and money spent performing safety preclinical and clinical trials and endless mountains of paperwork and years of back and forth with them.

Blame the FDA and... realize biology is a fucking mess. Specially with novel therapeutic vectors. We've rushed before in clinical trials that have maimed and killed humans.

I remember one of those, guess what happened? The field was frozen for almost 10 years. Nobody wanted to put money or political face on and have the same thing happen again.

So it takes years and years of mountains of paperwork and money and tests and more paperwork to prove safety first and then efficacy. I'd rather do most of these by compassionate use, but... I'm not in charge.

It takes about a generation to go from technological breakthrough to large scale deployment of these types of innovation.

There are a lot of nuts-and-bolts R&D, medical safety testing, manufacturing, supply, and logistics issues to consider when looking at how new discoveries will broadly impact society.

So, from the initial Crispr breakthrough to actually seeing the effects of this revolutionary tech, you are looking at 15 - 25 years for some of the most important innovations to be noticeable for those of us outside the laboratory.

If you say so

Yep. Far more concerned about the future inequality to come from crispr thsn the benefits..

Big pharma- put massive effort to cure minor genetic defects from low to middle income famalies. OR

-super engineer billionares children for huge cost and take government funding to steralize unwanted populations.

I think option 2 is much more likely unfortunately.

Yeah, pharmaceutical companies are extremely eager to make themselves obsolete. You’re right. What was I thinking?

Because of money, you know, fucktard?

This has always been true. Increasingly we are able to use biological modeling in virtual space to simulate trials as we did with the COVID-19 vaccine. In that instance bringing the development time down from 5 to 10 years to one year.

Remember exponential not linear

The short answer is yes. Eventually, as tech progresses and our knowledge base increases you could turn a bodybuilder into a zebra if you wanted.

That implies that it would be possible that an ex girl of mine turns into a horse and I have to admit that I fucked her. Why you gotta scare me like that?

CRISPR is actually very cheap, you can order a kit online yourself if you really wanted to.

Yeah and effectively modify genes? You can order iron too its not economocal for the average person to create steel and manufacture products. CRISPR is the very intro to actually applying this at any scale

The pipeline for distruttive treatments like these is much slower to reach the masses

Good, good, fix the code, fix the problem.

Then don’t read it.

What do you mean delivery delivery delivery mantra? Not familiar with the space

This is kind of a what it says on the tin case, basically one of the biggest problems being tackled in this field is successfully targeting and integrating treatments. As a result, even if this method turns out to be problematic down the line, it has a better than average chance of being significant.

!RemindMe 5 years

Burnt

Assuming crispr is involved there's only two marketable gene therapies rn. Supposed to be like 10 end of 23