Can someone with a more indepth understanding of the subject break down the order of magnitude here? How much of a real world impact does this increase in precision make?
My understanding is that the main benefit here is speed. Other techniques can help guarantee accuracy already, but they currently can only do this by slowing down the rate the DNA is modified
I may be way out of my depth, but until we can edit all the cells of a living organism, isn't CRISPR limited to working with pre-embryos/embryos? If so, other than creating a potential super race, what is it of benefit to the living individuals with "imperfect" genes?
Just to clarify, that is a real question, not rhetoric.
Gene editing has a lot of medical potential outside of the sci-fi utopia/dystopia stuff. Especially diseases caused by a straightforward well-known genetic cause [1], such as sickle-cell disease, might very well be permanently treatable in the next decade or 2.
There's also more far-out ideas that might be viable involving permanently affecting the bodies biological machinery to improve it, like adding extra insulin producing capability to cells in diabetic patients.
(Context: I have a PhD in genomics - but not necessarily an expert in this sub-field)
It is possible, but still difficult, to edit cells in-place. One example is the liver, a highly vascularized tissue (lots of blood vessels). This means that you can use a blood-based delivery method, e.g. blood infusion of CRISPR components + other compounds that allow these components to enter cells. Over time, more of the liver cells are edited and you can reach a reasonable proportion of the cells using this method using current techniques (40-90%). Note that for many diseases you would not have to reach 100% of the cells in order to have a positive effect on health.
There are a few labs working on better targeted delivery methods. In general the approach is to "wrap" CRISPR components in an envelope that is able to target specific cell-surface proteins for the cell type of interest.
From my reading of the article it sounds like they are using physical attributes to help the interaction, ie poking the single strand dna back into place at the time of editing.
I’m sure that’s an over simplification but biology always appears messy to me when compared to other sciences, at least when it is done right…
I look at biology as the results of a billion years of tinkering (evolution) as apposed to an engineer sitting down and designing something from the ground up. DNA is like a massive code base that has been modified for billions of years with no documentation or refactoring. Tons of duplicate code, genes that depend on completely unrelated genes, tons of stale unused code to wade through. Scientists are trying solves bugs while working through a billion years of technical debt. On one hand we ended up with a amazingly reliable and efficient pumps in our chests. On the other hand we have backs that constantly break down and fail.
Gene editing will enable us to eventually cure cancer!!
I've been rooting for a crash-program in this direction and halting all development of improvements to radiation and chemotherapy treatment to waste unnecessary spending of effort and time.
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[ 5.0 ms ] story [ 44.5 ms ] threadJust to clarify, that is a real question, not rhetoric.
Basically an auto transplant with marrow
There's also more far-out ideas that might be viable involving permanently affecting the bodies biological machinery to improve it, like adding extra insulin producing capability to cells in diabetic patients.
[1] https://www.ncbi.nlm.nih.gov/books/NBK132154/
It is possible, but still difficult, to edit cells in-place. One example is the liver, a highly vascularized tissue (lots of blood vessels). This means that you can use a blood-based delivery method, e.g. blood infusion of CRISPR components + other compounds that allow these components to enter cells. Over time, more of the liver cells are edited and you can reach a reasonable proportion of the cells using this method using current techniques (40-90%). Note that for many diseases you would not have to reach 100% of the cells in order to have a positive effect on health.
There are a few labs working on better targeted delivery methods. In general the approach is to "wrap" CRISPR components in an envelope that is able to target specific cell-surface proteins for the cell type of interest.
Fascinating, thanks.
I've been rooting for a crash-program in this direction and halting all development of improvements to radiation and chemotherapy treatment to waste unnecessary spending of effort and time.