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CRISPR and Gene Editing Treatment

D

deleted862527

    Anyone else find this new treatment exciting? Hopefully one day in the future gene-editing treatment will be a routine, alternative treatment for those with genomic/chromosomal abnormalities (multiple sclerosis, cancer, lupus, etc). I just saw this one story about this little girl who had leukemia, and it was very progressive and nothing could be done for her. However, she was one of the first to apparently have been used with CRISPR, where they changed the DNA of her WBC's so they can recognize and attack the cancer cells...

    Sorry about my nerd freakout over this...But I really hope we get to see more of this technology. Sometimes I hear of these "Medical Breakthroughs" on the news, and never hear about it again.
     
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    md-2020

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      CRISPR Cas9 appears to be very much the real deal, though its been around for a while but on hold due to ownership court cases and the fact that human germline editing is not yet allowed in the US.

      If you read the literature closely there are some issues with their error repair mechanisms as well, which over heritable traits may induce greater mutation rates. I wouldn't hold my breath for this to become commonly used anytime soon.
       
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        One of the main problems with using CRISPR clinically is it's been hard to efficiently infect enough of the problematic cells to make a worthwhile difference. For example, researchers have been able to take organoids of CF patients and cure them in vitro in pitri dishes using CRISPR, but there hasn't been a way to grant the virus access to the lung stem cells in vivo. You can infect respiratory tract cells but as soon as the tissue is turned over they are just replaced by new problematic cells since the virus does not have access to the progenitor cells.

        I think a more exciting and promising discovery that involves CRISPR is gene drives. Effectively we may be able to cure malaria and Lyme disease by destroying the pathogenic gene in the carrier host that allows transmission to humans. Therefore the disease still exists, it just can't be transmitted to/contracted by humans. Ideally this would not even affect carrier host populations, although I wouldn't mind if we accidentally wiped out all mosquitoes.
         
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        cactusman

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          Nah you're the only one who's hopeful about CRISPR and gene therapy.
          Then you must be one miserable, poor soul.
          Please know that I've done my best for months to hold back from sarcastic answers to SDN questions starting with "anyone else...?"

          "Has anyone ever picked a top 5 school over a full ride to a top 20-25 school?"
          "Did anyone become a doctor because of their parents?"
          "Has Anyone Ever Done "Caribbean Medical Schools"?"
          "Has anyone ever started an on-campus organization?"


          When online, I can only hold back my sarcasm for so long.
           
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          D

          deleted862527

            Please know that I've done my best for months to hold back from sarcastic answers to SDN answers starting with "anyone else...?"

            "Has anyone ever picked a top 5 school over a full ride to a top 20-25 school?"
            "Did anyone become a doctor because of their parents?"
            "Has Anyone Ever Done "Caribbean Medical Schools"?"
            "Has anyone ever started an on-campus organization?"


            When online, I can only hold back my sarcasm for so long.

            That's really pathetic to be honest.
             

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              Please know that I've done my best for months to hold back from sarcastic answers to SDN answers starting with "anyone else...?"

              "Has anyone ever picked a top 5 school over a full ride to a top 20-25 school?"
              "Did anyone become a doctor because of their parents?"
              "Has Anyone Ever Done "Caribbean Medical Schools"?"
              "Has anyone ever started an on-campus organization?"


              When online, I can only hold back my sarcasm for so long.
              Has anyone online ever held their sarcasm back forever?
               
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              md-2020

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                Crispr is too mainstream now.. It was cooler when I was one of the few writing about it but now everyone knows...

                Now you have the filthy casuals talking about Crispr using the headline "anyone else..."
                Do you even know what TALEN is kiddo? You're not a nerd, you're a basic beach.
                decom_Watch%20out,%20we%20got%20a%20badass%20over%20here!_56db525d43491.jpg
                 
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                thedarkniteeee

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                  Well you have to understand that gene editing already has been done; dates back to the 1990s with SCID (Sever Combined Immunodeficiency). The problem is that years later, alot of the original children who underwent the therapy died due to leukemia b/c of nonspecific gene therapy... the biggest thing to overcome right now is specific targeting (e.g. can we cut just that gene? and then even more specific can we specifically cut only one chromosome?)
                   
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                  Goro

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                    Anyone else find this new treatment exciting? Hopefully one day in the future gene-editing treatment will be a routine, alternative treatment for those with genomic/chromosomal abnormalities (multiple sclerosis, cancer, lupus, etc). I just saw this one story about this little girl who had leukemia, and it was very progressive and nothing could be done for her. However, she was one of the first to apparently have been used with CRISPR, where they changed the DNA of her WBC's so they can recognize and attack the cancer cells...

                    Sorry about my nerd freakout over this...But I really hope we get to see more of this technology. Sometimes I hear of these "Medical Breakthroughs" on the news, and never hear about it again.
                    It's the real deal, but the potential for abuse is highly concerning
                     
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                    BtmoreRavens18

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                      A few issues:
                      1. Massive off target effects that we're only just starting to get our head around
                      2. Vey difficult to target specific cell types/tissues which limits it therapeutic value
                      3. Some pretty terrifying possibilities if not regulated correctly - designer babies etc.

                      It's really useful in the lab though!


                      Sent from my iPhone using SDN mobile
                       
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                      Shirafune

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                        A few issues:
                        1. Massive off target effects that we're only just starting to get our head around
                        2. Vey difficult to target specific cell types/tissues which limits it therapeutic value
                        3. Some pretty terrifying possibilities if not regulated correctly - designer babies etc.

                        It's really useful in the lab though!


                        Sent from my iPhone using SDN mobile

                        Agreed. I think we are still way too far from practical clinical applications. CRISPR/Cas will have a bigger influence, at least in the nearest future, by opening so many doors in basic science. Medicine is going to benefit from a better, quicker understanding of pathophysiology than direct gene editing. The basic science of CRISPR/Cas is still in its infancy relative to the level required for mainstream, clinical use.
                         
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                        CommyO

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                          A few issues:
                          1. Massive off target effects that we're only just starting to get our head around
                          2. Vey difficult to target specific cell types/tissues which limits it therapeutic value
                          3. Some pretty terrifying possibilities if not regulated correctly - designer babies etc.

                          It's really useful in the lab though!sc


                          Sent from my iPhone using SDN mobile
                          1. Agree with point 1; site selection is vital for Crispr to perform as intended. Site selection could be improved with the use of sgRNA.
                          2. You have to consider that Crispr is still very new and further experiments will demonstrate it's therapeutic value. The word therapeutic carries a connotation that brings about hospital/clinic/etc. It's going to be scientists who implement it. I doubt physicians will get their hands on it within the next century.
                          3. Like all gene alteration tools, there are cons.

                          I know of quite a few labs who use CRISPR and it is very efficient for rapid knockout, deletion, or otherwise mapping genes.
                           
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                          BtmoreRavens18

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                            1. Agree with point 1; site selection is vital for Crispr to perform as intended. Site selection could be improved with the use of sgRNA.
                            2. You have to consider that Crispr is still very new and further experiments will demonstrate it's therapeutic value. The word therapeutic carries a connotation that brings about hospital/clinic/etc. It's going to be scientists who implement it. I doubt physicians will get their hands on it within the next century.
                            3. Like all gene alteration tools, there are cons.

                            I know of quite a few labs who use CRISPR and it is very efficient for rapid knockout, deletion, or otherwise mapping genes.

                            1. The sgRNA are of course necessary for CRISPR to have any selectivity at all but good site selection is far from sufficient to protect against off target. The Cas9n development may help, but ultimately this system seems to be quite promiscuous and can tolerate a lot of mismatch. Also, there does seem to be some off target effects just from putting cas9 into mammalian cells and this might take a long time to overcome.

                            You're right, it is fantastic in lab (although off target effects are very real!) OP was talking about therapeutic applications though, which is why I brought up 2 and 3.
                             

                            moggat

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                              A lot of labs are moving away from constitutively expressed Cas9 in cell lines and for mouse microinjection and switched to transient or inducible, which seems to limit off target effects much better!

                              Unfortunately, that's not useful for gene therapy lol, as viral vectors require integration by definition but I'm sure someone is working on a reversible/inducible or transient version. Obviously there are a host of other limitations, but I think that one will be improved within a reasonable amount of time.
                               
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