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Mutations causing anemia corrected in living patient, with no erros using CRISPR

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K

Kinitari

Black Canada Mafia
http://www.independent.co.uk/life-s...ue-could-revolutionise-treatment-9649983.html

Scientists have performed a “seamless” correction to a faulty gene behind an inherited form of anaemia using a revolutionary new technique in genome editing that could transform the treatment of many genetic diseases.

Two mutations in the haemoglobin gene of a patient with beta thalassemia – which can cause severe anaemia – were corrected without any errors using the Crispr technique of genome editing, the researchers said.

...

Crispr is a new way of editing the human genome with extreme accuracy and efficiency, and has generated intense interest across the world since it emerged last year. In this study it corrected the two inherited mutations of a Chinese patient’s haemoglobin gene with perfect precision, the scientists said.
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More at the link, CRIPSR is tearing shit up - it's making safe manipulation of genetic information possible at extremely low cost. This shit was only a thing last year, and people are going nuts over it. Very exciting time to be in genetics I imagine.
 
R

Raist

Banned
CRISPR is fucking awesome. It's gonna be a nobel prize fairly soon, I'm sure.

Thread title is a bit misleading though:

However, although the Crispr editing produced healthy red blood cells in laboratory dishes, the researchers did not transfuse them back into the patient – a critical step that would require further research as well as ethical approval, they said.
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“Although we and others are able to differentiate iPSCs into blood cell progenitors as well as mature red blood cells, the transplantation of progenitors into mouse models to test them has so far proven very difficult,” Professor Kan said.
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But the potential is definitely there.

I laughed at the typo on "errors", too :p
 
W

wetflame

Pizza Dog
This might be a stupid question, but where exactly is the gene that they're editing? It's not like there's one place in the human body that's broken and they go in and fix that one bit and the person's healthy again, right? Wouldn't the faulty gene be everywhere in the body? I obviously don't understand biology, haha.
 
R

Raist

Banned
wetflame said:
This might be a stupid question, but where exactly is the gene that they're editing? It's not like there's one place in the human body that's broken and they go in and fix that one bit and the person's healthy again, right? Wouldn't the faulty gene be everywhere in the body? I obviously don't understand biology, haha.
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In red blood cells. Well, immature ones, since mature red blood cells don't have a nucleus anymore. So they fix the gene in stem cells, and turn them into red blood cells.

This is mostly a proof of concept (likely coming from their own interest in that disease), because in practise it would be a bit tedious. Red blood cells don't proliferate and only have a rather short life cycle (3-4 months) so you'd need to periodically inject new healthy red blood cells.
 
Vlodril

Vlodril

Member
This is great news. Unfortunately its a few years out yet so i will probably not benefit from it but there are a couple of younger generations that will (there are less people these days with the disease due to the blood checks on pregnancy although some parents decide to keep the child anyway).

Good job science :)
 
P

Prezhulio

Member
Raist said:
CRISPR is fucking awesome. It's gonna be a nobel prize fairly soon, I'm sure.

Thread title is a bit misleading though:





But the potential is definitely there.

I laughed at the typo on "errors", too :p
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Amazing biotech (and it's not the only edit/delete/insert system out there even!) but this thread title is misleading, as you said. This "final step" isn't well understood for anything but plants (in a pretty different system not very translatable to mammalian use) and will be decades and decades of clinical trials.
 
L

Luigiv

Member
Oh cool. I have alpha thalassemia (the less serious cousin of beta thalassemia) I imagine this treatment can fix that too. I wouldn't mind a hemoglobin boost.
 
Y

Yagharek

Member
Just saying ... it's ethical to do lab trials on yourself. Maybe one day a researcher will test this out like the cure for h.pylori/stomach ulcers.
 
R

Raist

Banned
Prezhulio said:
Amazing biotech (and it's not the only edit/delete/insert system out there even!) but this thread title is misleading, as you said.
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Yeah, but I doubt talens and zfns are gonna go anywhere. Too costly and tedious to engineer.

This "final step" isn't well understood for anything but plants (in a pretty different system not very translatable to mammalian use) and will be decades and decades of clinical trials.
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Huh? What final step?
 
terrisus

terrisus

Member
Raist said:
I don't get the mention of plants.
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They've performed it on plants, but the step of performing it on humans is years away.

(According to the quote anyway, as far as how to process the quote. I have no idea about the actual situation)
 
terrisus

terrisus

Member
Amir0x said:
why, are you ill or very old? :(
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Well, I had cancer at 28, and a stroke at 30.
I'm only 31 now, but, I can't imagine making it long enough for them to have some way to prevent those from recurring before they do so. Not to mention whatever other fun surprises life has in store for me.
 
A

Amir0x

Banned
terrisus said:
Well, I had cancer at 28, and a stroke at 30.
I'm only 31 now, but, I can't imagine making it long enough for them to have some way to prevent those from recurring before they do so. Not to mention whatever other fun surprises life has in store for me.
Click to expand...

well...maybe your life exists on a frontloaded curve and it's clear skies from here on out? :(
 
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