Gene Replacement Therapy

Discussion in 'Science & Nature' started by K E Gordon, Oct 23, 2016.

  1. K E Gordon

    K E Gordon Very Well-Known Member
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    Gripping story this morning about children suffering from MLD a brain decaying disease that typically kills children before the age of 6. The family who had one child die from the disease and two others diagnosed found a clinical trial in Italy, and the other two kids are fine. However, gene therapy is not available yet in the US. They had some child die during clinical trials, and never restarted it. Now, the trial is closed in Italy. Even though this condition is rare, people need resources. I wonder if petitioning NIH would help? I lost focus on the story but I know the parents of another affected child were raising money. Maybe they were trying to get a private company to do this.
     
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  2. Corie Henson

    Corie Henson Very Well-Known Member
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    Gene replacement therapy sounds like stem cell. Obviously it is an expensive procedure. But how effective is it? I have this suspicion that gene replacement is the procedure done on one of the presidential candidate last May. It is just unfortunate that she was not able to sidestep stage 4 lung cancer.
     
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  3. Chrissy Cross

    Chrissy Cross Veteran Member
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    @K E Gordon, are the two kids fine because they had the therapy??
     
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  4. K E Gordon

    K E Gordon Very Well-Known Member
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    Yes, the two children who had the therapy seem to be doing well. Of course time will tell. I think it has been very effective with the two children who had the disease, because ordinarly kids who have this condition MLD, unpronounceable and unwritable real name, die before age 6 and one of them is a normal first grader now, riding bikes and playing soccer. They said they key is to find children before they are showing symptoms. You can see the entire story on CBS Sunday Morning. I love that show. It is the most unique one on TV if you ask me.
     
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    Last edited: Oct 23, 2016
  5. Chrissy Cross

    Chrissy Cross Veteran Member
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    It always hard when these trails are stopped. Usually it's funding or ethical problems.
     
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  6. Diane Lane

    Diane Lane Very Well-Known Member
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    @K E Gordon Were the parents aware that the kids had the condition because their other children had it? I would think unless you knew what to look for, and that there was some sort of predilection toward the disease in the family, it would be difficult to even know to get the child tested before symptoms were evident.
     
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  7. K E Gordon

    K E Gordon Very Well-Known Member
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    Yes, very astute of you @Diane Lane, they had an older child die of the disease, so they had the younger children tested, and they tested positive. The story reminded me a bit of Lorenzo's Oil. Anyway, I think we have to realize how blessed we are to have healthy children. The Philadelphia couple who had a child afflicted and are raising money for clinical trials, now it is too late for their daughter.
     
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  8. K E Gordon

    K E Gordon Very Well-Known Member
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    Well, I think in this case, was the death of a child in the study. It was what really made researchers lose their mojo.
     
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  9. Ina I. Wonder

    Ina I. Wonder Very Well-Known Member
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    @Chrissy Page do you mean today's episode. I tape it every Sunday, because most people know that I'm a morning person, so the program is invariably interrupted. I started watching it the first day it started, and I've not missed many. I find it a very educational program. :)

    I maybe I should try looking for single persons recipes. That sould weed out many of the gazilion recipes that popped up when I looked. :eek:
     
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  10. Chrissy Cross

    Chrissy Cross Veteran Member
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    #10
  11. Ina I. Wonder

    Ina I. Wonder Very Well-Known Member
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    Oooops! I did it again, and calling it a senior moment doesn't work too well here. :oops:

    I keep having trouble with the online connection today. So I guess I'm jumping the gun on my post a bit. :rolleyes:

    If that explanation works I'll duck now. :p
     
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  12. Chrissy Cross

    Chrissy Cross Veteran Member
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    I'll believe it, @Ina I. Wonder because I've had trouble all day. :)
     
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