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Thread: Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Disease

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    Administrator Islander's Avatar
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    Default Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Disease

    Jamie Ducharme
    April 18, 2019

    Researchers used an experimental gene therapy to develop a possible treatment for a rare and deadly immune disorder known as “bubble boy” disease, the National Institutes of Health (NIH) announced Wednesday.
    Because of a gene mutation, babies who are born with X-linked severe combined immunodeficiency (X-SCID) do not develop immune cells properly, leaving them highly susceptible to infections. The condition, which strikes up to one in 50,000 newborns, primarily affects boys and requires extreme measures to prevent infection. In one famous case, a boy with SCID, David Vetter, lived in a sterile plastic “bubble” until his death at age 12 — hence the disease’s nickname.

    Read more: http://time.com/5573015/gene-therapy...3a%22USA%22%7d
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    Veteran Member Mr. Wizard's Avatar
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    Default Re: Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Dis

    The article does not specifically refer to the type of gene therapy used to cure Bubble Boy. But, the CRISPR gene-editing technology has been growing in prominence because of its potential to cure many of these type conditions. CRISPR is not without controversy. Some worry that overzealous researchers will use CRISPR to do more harm than good. Certainly the potential to use CRISPR for evil exists, and this possibility shouldn't be ignored. Ultimately, I believe the benefits of CRISPR will prove too numerous to be stifled. However, the "ethics police" should remain vigilant to guard against producing "enhanced humans."
    Last edited by Mr. Wizard; 04-20-19 at 04:19 PM.

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    Default Re: Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Dis

    This was a topic that came up 35 years ago when I was teaching a college senior seminar on Great Issues in World Civilization. (Sounds intimidating, doesn't it)? One of the topics was genetic modification — we called it genetic engineering back in the 80s — and one subset of the topic was just this, the potential for genetic manipulation of unborn children. I remember acknowledging that it might have therapeutic applications, but beyond that the possibility of abuse was practically unlimited. I remember advising and cautioning my students that these topics would likely occur in their own lifetime and they should be prepared to give serious thought to the outcomes. And here we are, standing on the edge of that very cliff.
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    Veteran Member Mr. Wizard's Avatar
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    Default Re: Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Dis

    Last December, you posted an article here at HH about CRISPR babies, created by a Chinese researcher, which gave rise to a vibrant discussion. Bottom line: gene-edited babies isn't anything new. Was done before here in New Jersey a few years ago. Here's that thread:

    https://www.hawkeshealth.net/communi...ghlight=CRISPR

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    Default Re: Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Dis

    Thanks for the reminder, Mr. Wizard. You prompted me to do a search on "concerns around crispr" and this article thoroughly addressed overall concerns.

    CRISPR DNA editing may cause serious genetic damage, researchers warn

    https://techcrunch.com/2018/07/16/cr...earchers-warn/

    Even more specific is this one targeting 15 problems with Dr. He's experiment with the Chinese twins:
    The CRISPR Baby Scandal Gets Worse by the Day
    https://www.theatlantic.com/science/archive/2018/12/15-worrying-things-about-crispr-babies-scandal/577234/

    I think browsers treat Google searches differently. Safari shows only the search term(s) you requested; Firefox also displays a list of related items that might better fit your needs, like this. Nice, yes?


    • problems with crispr
    • crispr-cas9 human genome editing: challenges, ethical concerns and implications
    • crispr legal issues
    • crispr misuse
    • crispr unethical
    • crispr bioethics
    • benefits of crispr
    • is crispr ethical or unethical

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    Veteran Member Mr. Wizard's Avatar
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    Default Re: Gene Therapy Breakthrough Could Offer Treatment for Rare, Deadly 'Bubble Boy' Dis

    Wow. Very informative. Presently, most of the focus appears to be on the potential for harm. And, rightly so! But, ultimately, I believe the magic of CRISPR will outweigh the concern for harm. For example, CRISPR technology could prove beneficial for "congenital disorders," like heart conditions; Down syndrome; cerebral palsy; spina bifida, cystic fibrosis; Fragile X Syndrome, and so many more. Fragile X Syndrome is the most common "genetic" cause of autism. Given the the increasing numbers of autistic children, what parent would refuse the use of CRISPR technology if it meant freeing their child from a lifelong affliction like autism??? This genie is out of the bottle, and I don't see it being pushed back in. Having said all that, I still think the "ethics police" should be ever soooo vigilant.

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