Imagine Not Being Able To Breath Fresh Air Or Hold And

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Imagine not being able to breath fresh air or hold and feel the smooth touch of your parents. That’s what children with SCID’s face everyday from the day they are born. If not for gene therapy these children face life long isolation to survive. Today it has been widely reported that gene therapy offers a long-term treatment for a rare condition that causes children’s immune systems to fail. In the rare condition, known as Severe Combined Immunodeficiency (SCID), inherited genetic mutations mean that babies are unable to fight off infection, severely limiting their chances of surviving more than a few years. SCID’s are a rare genetic disorder estimated to affect between 1 in 200,000 and 1 in 1,000,000 live births (it is difficult to…show more content…
The child followed for the longest time, nine years, still had functioning immune cells, showing that gene therapy works in the long-term. Prior to gene therapy the only other option for children with the most common form of SCID was to receive a bone marrow transplant, which relied on finding a suitable match. This new technique, while not without risk, offers a credible treatment option in cases where a suitable donor cannot be found. The news also highlights the potential for using gene therapy to treat a number of other genetic conditions, although it should be emphasised that there is no guarantee that these diseases can be treated as successfully. In the two longer-term follow-up studies recently published in Science Translational Medicine, the underlying genetic defect was repaired in four out of six patients with Adenosine Demaninase-Deficient SCID, and 10 out of 10 patients with X-linked SCID. Immune cell production was restored, and the effects persisted up to nine years after therapy (the most recent point of measurement). The procedure produced minimal side effects, and patients could attend typical schools. Combining the results with the results of other studies shows that 30 patients with Adenosine Demaninase-Deficient SCID have been treated with gene therapy to date. All patients have survived and 21 have been able to stop enzyme replacement therapy. The authors of the London study conclude that
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