from the mutations-you-can-grow-out-of dept.
Submitted via IRC for takyon
The Broad Institute and MIT scientists who first harnessed CRISPR for mammalian genome editing have engineered a new molecular system for efficiently editing RNA in human cells. RNA editing, which can alter gene products without making changes to the genome, has profound potential as a tool for both research and disease treatment.
In a paper published today [October, 25, 2017] in Science, senior author Feng Zhang and his team describe the new CRISPR-based system, called RNA Editing for Programmable A to I Replacement, or "REPAIR." The system can change single RNA nucleotides in mammalian cells in a programmable and precise fashion. REPAIR has the ability to reverse disease-causing mutations at the RNA level, as well as other potential therapeutic and basic science applications.
"The ability to correct disease-causing mutations is one of the primary goals of genome editing," says Zhang, a core institute member of the Broad Institute, an investigator at the McGovern Institute, and the James and Patricia Poitras '63 Professor in Neuroscience and associate professor in the departments of Brain and Cognitive Sciences and Biological Engineering at MIT. "So far, we've gotten very good at inactivating genes, but actually recovering lost protein function is much more challenging. This new ability to edit RNA opens up more potential opportunities to recover that function and treat many diseases, in almost any kind of cell."
REPAIR has the ability to target individual RNA letters, or nucleosides, switching adenosines to inosines (read as guanosines by the cell). These letters are involved in single-base changes known to regularly cause disease in humans. In human disease, a mutation from G to A is extremely common; these alterations have been implicated in, for example, cases of focal epilepsy, Duchenne muscular dystrophy, and Parkinson's disease. REPAIR has the ability to reverse the impact of any pathogenic G-to-A mutation regardless of its surrounding nucleotide sequence, with the potential to operate in any cell type.
Unlike the permanent changes to the genome required for DNA editing, RNA editing offers a safer, more flexible way to make corrections in the cell. "REPAIR can fix mutations without tampering with the genome, and because RNA naturally degrades, it's a potentially reversible fix," explains co-first author David Cox, a graduate student in Zhang's lab.
Source: http://news.mit.edu/2017/researchers-engineer-crispr-edit-single-rna-letters-human-cells-1015
(Score: 1, Flamebait) by c0lo on Friday November 10 2017, @07:54AM (5 children)
Read: it's a potentially money maker treatment - let the fix "reverse" and then apply the expensive treatment once again... and again, until the patent can no longer by prolonged.
https://www.youtube.com/watch?v=aoFiw2jMy-0 https://soylentnews.org/~MichaelDavidCrawford
(Score: -1, Redundant) by Anonymous Coward on Friday November 10 2017, @08:17AM (1 child)
You are you suggesting it would be better not to have such a treatment?
Until base-pair editing becomes something a low-income "technician" can do for you, fucking with the master copy sounds like a recipe for disaster. Better to keep some level of indirection in place.
(Score: 1, Offtopic) by c0lo on Friday November 10 2017, @08:47AM
What I am suggesting: there's no incentive for them to reach a point in which a low-income "technician" can do it until the patent expires
Unless, of course, the Chinese get their own research going and F/O the US patent. It's highly possible/probable because, you see, Feng Zhang sounds as such a traditional American name.
https://www.youtube.com/watch?v=aoFiw2jMy-0 https://soylentnews.org/~MichaelDavidCrawford
(Score: 1, Informative) by Anonymous Coward on Friday November 10 2017, @12:47PM
Shame there isn't a mod for ignorant, know-nothing, impulse posting.
(Score: 2) by mrchew1982 on Friday November 10 2017, @01:55PM
I came to the comments to post exactly the same thing, because it degrades they will have you on the hook for recurring treatments.
On the one hand, research and drug approval are fsking expensive, and the greatest human motivator is greed, so no profit, no research. On the other hand, drug companies... At least in the USA... Are insanely profitable, seems wrong to take advantage of sick people to make obscene amounts of money. I wish that there was some kind of magical fix, government regulation in the form of parents seems to have created the problem in the first place.
(Score: 2) by All Your Lawn Are Belong To Us on Friday November 10 2017, @04:59PM
[Citation needed]. I read nothing in the release that says it's a treatment that must be repeated (at least, not any different from changing the DNA.) While you could be correct and it's an enzyme that directly affects translation and must be present whenever translation is made, it could equally be an enzyme which alters the RNA polymerase in such a way that it is permanent until it needs to be changed again. (Program, then reprogram, as opposed to constantly programming).
You could be right, but I see nothing in the summary nor TFA to substantiate what you say. Wish I had a subscription to Science but my school doesn't have it in its database apparently.
This sig for rent.