Editing human genetics - "New data on CRISPR treatment for blood diseases suggest cure is possible" by 1KIKI

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Editing human genetics - "New data on CRISPR treatment for blood diseases suggest cure is possible"

POSTED BY: 1KIKI
UPDATED: Monday, December 7, 2020 17:19
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Sunday, December 6, 2020 11:23 AM

Goodbye, kind world (George Monbiot) - In common with all those generations which have contemplated catastrophe, we appear to be incapable of understanding what confronts us.

Editing human genetics

Preliminary but ‘nothing short of great.’ New data on CRISPR treatment for blood diseases suggest cure is possible

Ten patients treated with a CRISPR-based gene-editing therapy for the inherited blood disorders sickle cell disease and beta-thalassemia have shown a consistent and sustained response with manageable side effects, according to interim results from two clinical trials reported Saturday.

Vertex Pharmaceuticals and CRISPR Therapeutics are jointly developing the one-time gene-editing treatment called CTX001. After being given the therapy by infusion, all patients in both studies have been free from symptoms of the diseases and have not needed blood transfusions. Although follow-up is still relatively short, the encouraging results raise hope that one-time cures for both blood disorders might be possible.

CTX001 is the first and most advanced effort to use the Nobel Prize-winning CRISPR/Cas9 technology to provide functional cures for inherited diseases.

https://www.statnews.com/2020/12/05/preliminary-but-nothing-short-of-g
reat-new-data-on-crispr-treatment-for-blood-diseases-suggest-cure-is-possible/

Read more at the link. Now, this CRISPR treatment only edits cells in the bone marrow (as far as anyone knows), so any children of these patients can still inherit their parents' faulty genes. And CRIPSR on fertilized eggs has so far produced many unwanted gene changes. Eventually however, someone will successfully tinker with inheritable human DNA, for better or worse.

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Monday, December 7, 2020 11:52 AM

SIGNYM

I believe in solving problems, not sharing them.

Didn't some rogue Chinese scientist claim to have CRISPER edited genes at the fertilized egg stage, giving rise to two (presumably) healthy babies?

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Pity would be no more,
If we did not MAKE men poor - William Blake

#WEARAMASK

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Monday, December 7, 2020 12:41 PM

SIGNYM

I believe in solving problems, not sharing them.

Ah... it's as I thought.

The process isn't one of "simple" tranfusion: They have to harvest patient stem cells (i.e go in and core out a big sample of bone marrow), edit the stem cells, kill off the paient bone marrow, and re-introduce the edited stem cells.

Not a simple, risk-free procedure! This is very similar to how they treat people with various forms of leukemia.

But I suppose the progress is that the good cells are introduced by infusion and captured by the bone marrow, where they start reproducing, so the researchers don't have to do anything fancy like virally-introducing the edited genes into multiple organs of the body.

I wonder how that might work for other diseases ... say, metabolic diseases, where a specific enzyme isn't produced. As long as the enzyme can be circulated thru the blood (and not have to be endogenously produced in each cell) maybe they can grab liver cells and introduce positive stem cells into the liver and make it the chemical factory for the rest of the body.

Just a thought.

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Pity would be no more,
If we did not MAKE men poor - William Blake

#WEARAMASK

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Monday, December 7, 2020 5:19 PM

1KIKI

Goodbye, kind world (George Monbiot) - In common with all those generations which have contemplated catastrophe, we appear to be incapable of understanding what confronts us.

Re that Chinese scientist - I don't know at what stage he supposedly edited the embryos, but (non-human) research is finding that if you edit at the fertilized egg stage, so that all subsequent cells have the edited genes, development goes terribly askew, and they find those edits where they don't belong.

It does seem in theory perfectly possible to modify cells using CRISPR-Cas9 and reimplant them to be factories for the body. I'm just not sure how tissue-specific the cells have to be. For example, if you're trying to repair a bone marrow problem, will liver cells be suitable?

To be sure, limited gene therapy and reimplantation of altered cells to correct deficiencies has already been done, but the genes are inserted by adenoviruses at random. https://en.wikipedia.org/wiki/Gene_therapy If you add a good gene to a genetic set with a deficient gene, the good gene can pick up the slack for the bad gene. So you can correct what are essentially recessive genes.

The nice thing about CRISPR-Cas9 is that you can edit the bad gene in place (in theory). You can correct a dominant bad gene.

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