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a point that small children are not excluded from the harsh reality of life time illnesses. For example, a number of young children were diagnosed with a metabolic disease called metachromatic leukodystrophy. Children diagnosed with this disease lack a critical enzyme causing them to gradually become paralyzed and eventually dying within a couple years. Gene therapy injections are a beacon of hope for the three children who were diagnosed with metachromatic leukodystrophy”, After two years of receiving one injection the children have been producing enough enzymes to keep their brain and spinal cord working normally. This has given these children the chance to live out close to normal childhoods, giving them the options of being in sports, running around with friends, or even simply playing in the park on a warm and sunny day. Gene therapy gave these children back their right to their own pursuit of happiness and has been nothing but beneficial to them. In the next paragraph i would like to talk about hereditary bleeding disorders that can be cured with protein injections. These injections have been developed and classified under the medical class of gene therapy. People with hereditary bleeding disorders often don't get to experience the many privileges that most of us take for granted such as simply running a marathon, skate boarding, and as odd as it seem they don't get the privilege of not worrying to much about a minor nose bleed.
For example, people with “Hemophilia B” have to worry about those “minor” cuts and bruises because they lack a protein called Factor IX, an essential part of the blood clotting process. In simpler terms people with this hereditary bleeding disorder can die from what seems like a minor thing to others. “Hemophilia B” is caused by a mutation in the gene for protein production that is carried on the X chromosome, meaning the disease is exclusively for men, about 1 in 30,000 individuals inherit the mutation. Patients diagnosed with this illness can turn to protein injections to improve their bodies Factor IX production (disabled protein). One patient who received a one time infusion saw a positive increase in his production levels for more than 18 months after only one treatment. This proves to be beneficial to society by showing that even the most rare cases can be helped through the medical class of gene therapy. Now in the next section I'd like to talk about how i would like to talk how gene therapy can improve not only patients lives but everyone's as a
whole. As people we often don't concern ourselves with the struggles that other people face because the truth is we are selfish creatures. But what if your sister was diagnosed with a factor VII deficiency? Meaning she would never be able to give birth for she would surely die from bleeding out. Or what if a baby suffers from the same factor VII deficiency and dies due to bleeding in the brain. What about remembering all the fun childhood memories you and your cousin created and having to share them at their funeral because “Metachromatic leukodystrophy” claimed them. Even household pets can be claimed by genetic mutations. My point being is gene therapy is a treatment option that has been proven to be a positive option to cure all sorts of diseases or conditions and has had many success stories to prove the benefits. Gene therapy gives hope to all people, even if you're young, old, or somewhere in between. Gene therapy is overall beneficial to everyone, either directly or indirectly...
If faced with a life or death situation, would you turn down the chance to be cured and saved? Or would you be thankful that medicine has evolved into what it is and all it has to offer? I believe the good out shines the bad when it comes to gene therapy and I truly believe that it is absolutely one of the most beneficial types of medicine out there. The next time you feel a type of medicine is “monstrous” or “horrific”, just remember people once feared the idea of gene therapy and now it is turning into one of the most life saving forms of medicine.