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Cells edited using CRISPR–Cas9 injected into a person for the first time

nature.com

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Re: Cells edited using CRISPR–Cas9 injected into a person for the first time

#31

Post by gajjanag »

leggomylibro wrote: post #15
dave_sullivan wrote: post #13

Earlier quoted context omitted.

> it would probably make more sense to just knock out the activated oncogene(s) in the patient's cancer I've been wondering about that for the past couple of years, since I heard of CRISPR basically. Who is working on this aspect most seriously right now? Is there any interesting published progress? I understand it will take a while to make real progress, but mostly I'm just curious as an outsider to the industry.

I don't think anyone is; it's still too far out there. I'm sure that there's plenty of research building towards it, but I couldn't name any names. I'm also an outsider to the industry, but CRISPR is actually a simple protocol that doesn't require much advanced equipment, and I've been looking into using it for some home science projects. The problem as I understand it is that right now, CRISPR requires some manual s…

> CRISPR requires some manual steps in the lab to make it work well. You can design the protein and guide RNA and spacers until you're blue in the face, but if you can't find a specific enough cutting site on the genome, or one that is close enough to your target, or something like that, you'll wind up with a lack of specificity in that cut, which can lead to unwanted mutations.

I am not a biologist, but nevertheless I found out something interesting today regarding the problem of designing good guide RNA via machine learning.

The work that I learnt about today addresses 2 key questions via machine learning approaches: off-target effects of CRISPR (http://biorxiv.org/content/early/2016/10/05/078253) and optimization of the guide RNA sequence to maximize on-target activity and minimize off target effects (http://www.nature.com/nbt/journal/v34/n2/full/nbt.3437.html).

I learnt of this stuff from one of the authors who works at MSR New England (http://nicolofusi.com/).

Also of possible interest is their project page https://www.microsoft.com/en-us/research/project/crispr/, where they are planning to release a cloud-based service for end-to-end guide design by incorporating the approaches developed in the above work.

Re: Cells edited using CRISPR–Cas9 injected into a person for the first time

#32

Post by ihodes »

dave_sullivan wrote: post #13
leggomylibro wrote: post #12

So this title is a bit misleading; something like, "cells edited with CRISPR injected into a person for the first time" would be better. While CRISPR is promising for topological treatments, that's not what happened here. The team took white blood cells out of a patient's body, used CRISPR to knock out a gene which suppresses immune response, then injected those same cells back into the patient, hoping that they woul…

> it would probably make more sense to just knock out the activated oncogene(s) in the patient's cancer I've been wondering about that for the past couple of years, since I heard of CRISPR basically. Who is working on this aspect most seriously right now? Is there any interesting published progress? I understand it will take a while to make real progress, but mostly I'm just curious as an outsider to the industry.

Startups working on this (with credible founders, i.e. inventors of CRISPR) include Editas Medicine, Caribous Biosciences, and CRISPRx. Look out for Doudna, Qi, Charpentier, Elledge, and Church. Also see J. Listgarten for computation approaches to improving CRISPR/CAS9.

This isn't ready for in vivo use yet due to off-target effects (CAS9 is a very permissive protein that often binds to the wrong part of the DNA).

In the US, Carl June's group at U Penn is working on editing adoptive, engineered CAR T cells for cancer therapy, going into clinical trials. They're probably bummed to have been scooped (first to do an adoptive CRISPR engineered T cell transfer), though their work is certainly more clinically relevant than Lu's.

Re: Cells edited using CRISPR–Cas9 injected into a person for the first time

#33

Post by ihodes »

nonbel wrote: post #14

>"The researchers removed immune cells from the recipient’s blood and then disabled a gene in them using CRISPR–Cas9, which combines a DNA-cutting enzyme with a molecular guide that can be programmed to tell the enzyme precisely where to cut. The disabled gene codes for the protein PD-1, which normally puts the brakes on a cell’s immune response: cancers take advantage of that function to proliferate. Lu’s team then…

It's certainly the former; CRISPR is used to mess with a gene, not kill a population of cells. This isn't what the trial is looking to validate.

Re: Cells edited using CRISPR–Cas9 injected into a person for the first time

#34

Post by kleigenfreude »

https://en.wikipedia.org/wiki/CRISPR -> Cures Disease

Eventually...

https://en.wikipedia.org/wiki/CRISPR -> https://en.wikipedia.org/wiki/Gattaca

And...

https://en.wikipedia.org/wiki/CRISPR -> https://en.wikipedia.org/wiki/Gremlins_2:_The_New_Batch

I hope we cure a lot of diseases before we get into the bad side of genmods.

Re: Cells edited using CRISPR–Cas9 injected into a person for the first time

#39

Post by Ralfp »

dharma1 wrote: post #38

I have a feeling this won't stop at medical use cases

Most of technology is neither good or evil, and has usages in both civilian and military space. That is how the things are with technological progress. Machine learning may be used to find corelation between gene mutations and cancer, but it may also be used to put missile into unsuspecting enemy.

Roman road system allowed for flow of goods and people within empire on scale never before seen in acient world, but also for their legions to strike empire's enemies.

Re: Cells edited using CRISPR–Cas9 injected into a person for the first time

#40

Post by andygates »

guelo wrote: post #20
leggomylibro wrote: post #12

So this title is a bit misleading; something like, "cells edited with CRISPR injected into a person for the first time" would be better. While CRISPR is promising for topological treatments, that's not what happened here. The team took white blood cells out of a patient's body, used CRISPR to knock out a gene which suppresses immune response, then injected those same cells back into the patient, hoping that they woul…

The headline seems fine to me. Your complaint seems to be that you think "CRISPR gene-editing" should only refer to editing of cells while they are still inside a live body. But I don't see why that specific type of therapy should claim full ownership of the term "CRISPR gene-editing". Grammatically, any therapy that uses CRISPR modified genes would qualify.

Watch the low-quality comments for an illustration of how to misread that headline as "We edited a human! I want wings and a giant pulsating brain!" /s
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