Brian Madeux had Hunter’s syndrome, an inherited, rare genetic disorder caused by a missing or malfunctioning enzyme (“MPS II”). As a result, Madeux underwent more than two dozen operations (Marchione, 2017). A treatment that consisted of weekly doses of the missing enzyme was available, but it would cost more than 100,000 dollars per year and could potentially lead to brain damage. So, in November 2017, Madeux decided to undergo gene therapy, making him the first person in the United States to have a gene edited inside of his body. When considering whether gene editing can result in cures or is just scary science, it becomes evident that gene therapy is a revolutionary new way to produce cures due to the number of lives it can impact.
Gene editing used to seem like science fiction, but now technological breakthroughs have made gene therapy a reality. Zinc-finger nucleases, or ZFNs, were discovered over 20 years ago and have been used multiple times to save lives such as Brian Madeux’s (Klug, 2010). With the advent of CRISPR, gene therapy has only gotten better. CRISPR, which can target and subsequently edit specific stretches of a genetic code, is the cheapest, easiest, and fastest form of gene therapy (Lockyer, 2016). Researchers at the Center for Genomic Engineering have found CRISPR to be 99% accurate (Kim et al, 2017). As a result, gene editing, mainly through CRISPR and ZFNs, has become a cost effective and quick way to cure diseases that previously required complicated treatments.
Unintended consequences are frequently scrutinized as a reason to scale back on gene editing. However, recent research has shown that ZFNs can be applied effectively for human gene therapy (Carroll, 2008). Advancements in the technology have resulted in minimizing the toxic side effects and large insertions have been conducted with much success. CRISPR is so accurate that doctors are comfortable with using it to experiment on genetic diseases (Ma et al., 2017).
The potential of this technology is growing at a rapid pace. For instance, scientists have used CRISPR to remove the DNA of HIV virus from mouse cells, effectively removing the virus from the animal (Yin et al, 2017). Although there are ways to treat HIV, the treatments tend to be ineffective and may have unintended consequences (Cihlar & Fordyce, 2016). In addition to HIV, CRISPR has been shown to have the potential to cure various ailments such as lung cancer, Duchenne muscular dystrophy, and Huntington’s disease (Yi & Li, 2016; Nelson et al., 2016; Yang et al., 2017). Furthermore, CRISPR even results in the desired trait passing through generations at a rapid pace (Lockyer, 2016). This means that gene therapy can even treat familial genetic diseases. Gene editing provides a simple and effective way to cure diseases and represents a crucial key in the future of medicine.
Gene editing is not limited to humans, but is also an efficient tool on disease-inducing insects. For instance, malaria, a disease that killed nearly half a million people in 2015, is passed on by mosquitos (“10 facts”, 2016). Scientists have discovered an efficient method of removing the malaria gene from mosquitos by using CRISPR (Gantz, 2015). The gene editing of the malaria gene resulted in 99.5% of offspring acquiring the mutation of the malaria vector. The study states that this method could play a key role in eliminating malaria around the world.
Brian Madeux was just the first of many to be saved by gene therapy. Through technological advancements and continuing medical research, gene therapy has become the cure of the future.