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  1. Gene Therapy Reverses French Teen’s Sickle Cell

    March 7, 2017 by Andrea
    Photo: OpenStax College [CC BY 3.0], via Wikimedia Commons

    Photo: OpenStax College [CC BY 3.0], via Wikimedia Commons

    No doubt your Twitter feed has been flooded with the news that recently broke about a French teenager whose sickle cell disease has been reversed due to gene therapy. Read on to find out how it all happened.

    Until about 15 months ago, the then-13-year-old French boy (whose name has not yet been released) had to return to the hospital monthly for blood transfusions to treat his sickle cell. And as the BBC reports, this boy's internal damage was so bad, he'd already had his hips replaced and his spleen removed.

    Then, doctors had the idea to try an experimental treatment on the boy, in which they would remove his bone marrow and genetically alter it, theoretically correcting the gene mistake that causes sickle cell. "By using a virus to insert genes for the correct form of [hemoglobin protein] into the bone marrow ... researchers have been able to restore the elasticity to the patient's blood," explains Science Alert. Afterwards, the bone marrow was returned to the patient's body to see if, in fact, the gene therapy would prompt the marrow to generate normal red blood cells.

    It worked, and so far, so good.

    Since the teen's operation, he's shown no signs of SCD whatsoever, and as a result, his hospital visits and medications have ceased. While far from a cure, though -- since this procedure has only been done, albeit successfully, on one person -- it does show how gene therapy can potentially be the key needed to unlock a true cure.

    Even if this is the case, the BBC notes another potential barrier -- the fact that the majority of sickle cell warriors live in African countries, and therefore do not have easy access to expensive gene therapy procedures, such as this. So, besides conducting more trials on many more patients, researchers will also need to discover a way to reduce costs, so that if a cure is indeed discovered, it can actually be used where it's needed most, to help as many millions of people as possible.

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