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What are the benefits of gene therapy?

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Quick Answer

Though still experimental, gene therapy fixes genetic defects and potentially reduces the need for drugs, radiation or surgical intervention. Replacing bad genes with good ones may eventually cure Parkinson’s, Alzheimer’s, cancer and many other diseases.

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Conceived in 1972, there are two kinds of gene therapy: somatic cell gene therapy and germline therapy. Somatic gene therapy repairs or replaces genes and cannot be inherited. However, germline therapy changes genes in reproductive cells and can be passed on to future generations.

In 2011, the New England Journal of Medicine reported that researchers had inserted a gene into several hemophiliac patients that boosted their ability to clot blood. At Cedars-Sinai Heart Institute in Los Angeles, California, genes injected into a small portion of a pig’s heart temporarily regulated its heartbeat. Further experiments may lead to human pacemakers.

However, people have ethical concerns about this treatment. Many are troubled with the idea of scientists playing God. Furthermore, if only the rich can afford gene therapy, this threatens to widen the gap of inequality.

Proper regulation is also an issue, as there have been unchecked, undocumented deaths and adverse side effects. Teenager Jesse Gelsinger died in September 1998 after gene therapy for a rare liver disorder lead to the failure of several organs. The Food and Drug Administration suggests that Gelsinger was not properly informed of previous patient side effects, as well as deaths in animal experiments.

Procedural risks are another concern. Proper procedure requires enormous precision and accuracy as well as complete research, as death can occur from a single mistake.

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