7 ms·
Gene therapy (and CRISPR) is ultimately going to eliminate most if not every ailment/issue/genetic problem that we have, including aging and eye problems. Very
by mandude 9y ago
Gene therapy (and CRISPR) is ultimately going to eliminate most if not every ailment/issue/genetic problem that we have, including aging and eye problems. Very thankful we are reaching the point where we can go into our cells and fix ourselves.
- revelation 9y agoThat was the plan from the start, no? I was pretty disappointed to learn that most drug research is essentially mass testing all kinds of molecular agents that happen to show some beneficial effect for a problem at hand. It seems just too simple a method.
- iplaw 9y agoThat is rapidly changing to a more targeted approach. However, don't downplay the importance of the shotgun approach. In the broadest sense, it gave us Viagra for ED (common reported side effect in an unrelated trial), Aleve (intended to be a hangover treatment you'd take the night before), ... With computer and analytical modeling, it's a much easier process to identify a problem and reverse engineer a treatment.
- ythn 9y agoSeems too good to be true
- warent 9y agoFortunately in the late 19th century so did the prospect of Moon exploration and arrogated human knowledge in our pockets. But it's pretty standard now
- omarchowdhury 9y agoMoon exploration isn't standard, but I get your point.
- warent 9y agoAggregated* not arrogated.
- pm90 9y agoTo be fair, they probably said the same about penicillin when it was discovered.
- quux 9y agoAnd in a way penicillin is too good to be true, now that bacteria are widely resistant to it and many other antibiotics.
- omarchowdhury 9y agoDoesn't erase the benefit it has had up to this point.
- eliben 9y agoPenicillin probably saved at least tens of millions of lives, and other antibiotics even more. Yes, some strains of bacteria are developing resistance, but even today antibiotics are saving many many lives every day.
- a_imho 9y agoCan we crispr resistant bacteria to be susceptible to penicillin/antibiotics again?
- ccozan 9y agoInteresting idea. So you would first infect voluntarily with a custom virus that targets the bacteria by using CRISPR to remove the DNA strand of the resistance, then simply use the usual antibiotics to kill it.
- mathw 9y agoAt that point couldn't you just make a custom virus which kills the bacteria, so it happens in one step?
- erklik 9y ago
- tejtm 9y agoI do agree, but that is just the good news. In other news although we can harbor optimistic thoughts for scientists, medical professionals, lawmakers and maybe even politicians all around the globe to do the "right" thing to preserve human life in a currently recognizable form, but there is not a force on heaven or earth that is going to stop parents from trying to give their children every advantage.
- return0 9y ago> to do the "right" thing "right" according to whom? nature itself doesn't believe that
- mtempm 9y agoDespite academic/political/military/banking/corporate alignment against fundamental morals, "right" appears to be in agreement with nature/natural selection. The States had a more "right" society founded om constitutional and civil rights that was a big part of its climb from an English colony to the world power in less than 150 years after the constitution was written. The reason people probably have strong convictions about right or wrong is most likely because of nature, not despite it. Groups that had this genetic trait built tribes, societies that had greater success in the long term. In the short term, individuals, or even groups of individuals, can be successful by acting amorally, but this comes as a cost to their group's long term success--the cancer analogy. Right now there is limited competition between groups in the world. Most of what we should be competing against is the coming extinction event when something else out there gets a whiff of all the artificial elctromagnetic radiation. Probably the reason we hear so little in the cosmos is because it is not a very competitive strategy.
- deleted 9y ago[deleted]
- golergka 9y ago> academic/political/military/banking/corporate alignment against fundamental morals "fundamental morals"? What are those and where the hell do you get them from? > The reason people probably have strong convictions about right or wrong is most likely because of nature, not despite it. Groups that had this genetic trait built tribes, societies that had greater success in the long term. Actually, couldn't agree more. But people do have _different_ convictions about right or wrong.
- dekhn 9y agoI dedicated a significant fraction of my life to the idea that gene therapy would become a viable medical treatment. It seems unlikely to really become a high-impact treatment. Being able to change DNA in cells is one thing; actually being able to show your treatment does what it's supposed to and didn't have negative effects is hard. There are a limited number of diseases where gene therapy should work great, but there are a wide range of others where it won't, until solve multiple grand-challenge class problems.
- tropshop 9y agoWould you elaborate on what makes a disease a good fit and these grand-challenge class problems?
- dekhn 9y agoA disease is a good fit if you can just inject the therapy into a localized region (say, the eye, or an organ) and the treatment works for a reasonably long period of time (months+). Typically another requirement is that the target is a defective gene where the phenotype can be repaired through addition of a "corrected" form of the gene, without the defective gene needing to be removed. This is the case in X-linked retinitis pigmentosa, where the genetic cause of the disease has been understood for some time, it's relatively simple underlying mechanism (so we think), and you can deliver the medicine to the retina with periodic injections. Treating a disease where you have to remove an inserted retrovirus from a large number of freely circulating or "hidden" cells (which is the case in HIV) is far more challenging- you need a way to recognize the cells of interest, access all of them, and get 100% transversion. All without causing negative side effects.
- gmarx 9y agoWe learned early on that one of the difficulties in eliminating HIV is that it hides in the nervous system and can reemerge at any time. This article show promise in that it can reduce HIV viral load during active shedding. I think it is less likely that it could ever eliminate HIV entirely (i.e. cure)
- maverick_iceman 9y agoEvidence?
- mandude 9y agohttps://www.researchgate.net/publication/315775073_In_Vivo_Excision_of_HIV-1_Provirus_by_saCas9_and_Multiplex_Single-Guide_RNAs_in_Animal_Models https://www.researchgate.net/publication/315775073_In_Vivo_E...