http://www.nature.com/nbt/journal/vaop/ncurrent/full/nbt.2884.html

We demonstrate CRISPR-Cas9–mediated correction of a Fah mutation in hepatocytes in a mouse model of the human disease hereditary tyrosinemia. Delivery of components of the CRISPR-Cas9 system by hydrodynamic injection resulted in initial expression of the wild-type Fah protein in ~1/250 liver cells. Expansion of Fah-positive hepatocytes rescued the body weight loss phenotype. Our study indicates that CRISPR-Cas9–mediated genome editing is possible in adult animals and has potential for correction of human genetic diseases.

an injection to change you at a genetic level

really is remarkable, the potential for treating diseases is huge

obviously will take a while to get 100% of cells (or as close to as poss) but the same treatment could be carried out on humans pretty much immediately and have a very positive effect.

sadly Ive not seen it reported anywhere, the media’s moral turpitude and crippling inability to understand science would only reduce it to a series of tragic headline grabbing soundbites anyway

an injection to change you at a genetic level

That’ll be the next level of doping, then. 🙁

Thought you were going to mention Turkey…

Was on the Today Programme yesterday morning.

And it’s superb news. Looking forward to its application sooner rather than later….

pondo – Member

That’ll be the next level of doping, then.

and impossible to test for?

unless you kept a baseline copy of someones genome, ideally from birth

http://en.wikipedia.org/wiki/List_of_genetic_disorders

MMMM torn between the good it could achieve and the fear of what humans will do with eugenics in an injection
Was Radio 4 not about Yeast as that was what I heard yesterday

the yeast one got more headlines as that sounds a bit more PLAYING GOD(tm) creating an entire choromosome

There was a program about this on Radio4 this morning.

Opinion from the experts on genetic disorders was that;

1) given current technology the chances of manipulating only a faulty gene in a genetic disorder and having no other side effects is fairly small.

2) you’re better off testing embryos/fertilised eggs in ‘at risk’ situations prior to implanting them.

They did say that all births in the US where there is a suspected genetic disorder are now tested and a resulting full genome analysis is available within 36 hours of the birth.

We’ll all end up looking like this:

I’ve got a teddy bear like that!
He’s called Zaphod Beeblebear…

Paul McCartney seems to be getting some use out of the head on the left in that pic.

…and has potential for correction of human genetic diseases.

The challenge for the media and the public at large is the perception that all day every day scientists are carrying out research that has potential to do X, Y and Z. hen it’s potential rather than deliverable, it’s hard not to default to headline-grabbing based on the agenda-du-jour.

I think it is tremendous news, especially since my son has a genetic disorder, but I can understand why this is not a mainstream headline…

…yet.

wwwawas

Im no expert but I am trying to use this technique at the moment in cultured human liver cells

1) Id disagree about off target effects- that technique is alreay superceeded, they only saw a very low % of off target effects and only in non coding regions and the newer version of the same technique reduces the % to as near to 0 as you can be

2) probably should sequence the genomes of all foetuses, though that in itself can carry risks but really you are talking about IVF for every conception!

I believe everyone should be sequenced at birth, but then you have the issue of security of info

kimbers I didn’t hear it all but it’s here;

http://www.bbc.co.uk/programmes/b03z9k48

I really don’t have the knowledge to present any more info than I have already so I’ll probably stop contributing after this post!

CRISPR gene editing is such an impressive technology – but presumably the biggest hurdle in most cases will be efficient delivery to your target cell type.

My mistake. It was about yeast.

And that’s why I’m not a scientist – no attention to detail!

youre right martinhutch, the naoparticle idea is agood one, – went to a talk recently about how the fields of MRI scanners can be so accurately controlled as to deliver them(nanoparticles) to specific locations in the body

local injection will have to do for now though

I gave up in the first sentence. Is there a translation?

Boffins have found a clever way to substitute bits of the genetic code in your cells for new versions. If you have a disease caused by a dodgy gene(s), in theory at least, this technique might one day help cure it or alleviate the symptoms.

At the moment they’ve used it to treat, apparently successfully, a genetic disorder affecting liver cells. It may be the first time that this has been achieved in an adult animal.

However, we are a fair way from using it safely in humans, and further away still from using it to treat a variety of more common genetic disorders, as you still have to get your gene-swapping technique into the right cells in the right part of the body.

thanks

wwwawas

Im no expert but I am trying to use this technique at the moment in cultured human liver cells

1) Id disagree about off target effects- that technique is alreay superceeded, they only saw a very low % of off target effects and only in non coding regions and the newer version of the same technique reduces the % to as near to 0 as you can be

Kimbers, linkies to your published papers mate? Would love to get a bit of work experience with you… one day… if this is the kind of work that you are into.

MMMM torn between the good it could achieve and the fear of what humans will do with eugenics in an injection

Ahhh ethics, always brilliant at being a buzzkill.

That’ll be the next level of doping, then.

And it should be totally legal… running can become the next formula one….instead of Mclaren vs Ferrari we can have GSK vs AstraZeneca. Bring on the genetically modified, microchip enhanced humans. 😈

However, we are a fair way from using it safely in humans, and further away still from using it to treat a variety of more common genetic disorders, as you still have to get your gene-swapping technique into the right cells in the right part of the body.

Yeap, if the track record for safety when it comes to things like stem cell therapy is anything to go by then we probably have a few decades before any therapy derived from this comes to market.

The RNA editing info has regularly been in the press for a while. It has huge potential but as usual practical things like genomic variation will cause a few problems. It reminds me of the hype surrounding viral gene therapy many years ago. That didn’t work out so well and people are still trying to figure out whether it’s got a place in clinical medicine.

Sequencing IVF embryos and prenatal sequencing……… it’s already been done………. it’s also hugely regulated in the UK ……….

Paul McCartney seems to be getting some use out of the head on the left in that pic.

🙂

Almost lost a mouthful of biscuit…

you still have to get your gene-swapping technique into the right cells in the right part of the body

pardon my ignorance but apart from speedy results why do you have to target the cells, if its genetic abnormality that needs fixing why can’t you change the lot?

Delivery issues are problematic with lots of these techniques. It’s difficult to get the CRISPR system into lots of cell types/tissues. That’s mainly why most techniques aim to target peripheral blood targets.

donk – each cell has its own copy of the genome, although only those that express the protein you are interested in need to be targeted, for example to change your eye colour youd have to deliver it to your eyes etc

if you want the change to be passed on to your offspring youd have to target the gonads

Damnit Kimbers! Can I come and work for you?