The advent of CRISPR/Cas9 genome editing is completely revolutionizing the way we approach scientific problems. This technology provides a relatively simple gene-editing platform to modify entire genomes. Although CRISPR holds great promise in transforming the way we approach human diseases, it remains largely unregulated because of the outdated and restrictive regulatory scheme for biotechnology in the United States (Claremont Journal of Law and Public Policy).
While scientists are racing to unleash the therapeutic potential of CRISPR genome editing, they are yet to confront the dialogue surrounding potential ethical, societal, and safety issues regarding gene -editing technologies. This stark disconnect was brought to light this past November at the Second International Summit on Human Genome Editing on the progress of emerging gene therapies when a shocking announcement was revealed. He Jianku, a Chinese researcher, announced that he had genetically edited twin babies (that had been brought to term) so that they were resistant to future infection with HIV. There was an immediate outcry from scientists around the world because Jianku had allegedly violated ethical norms and his patients’ safety (Marchione). In order to prevent another CRISPR controversy, I propose that we should develop a renewed regulatory scheme for gene editing technologies to (1) standardize gene editing protocols and (2) focus on incentivizing ethical research using human embryos to develop innovative, lifesaving treatments.
Clustered Regularly Interspaced Repeats (CRISPR) are the hallmark of bacterial immune systems. Scientists have re-engineered this system, consisting of a single molecule that targets a gene of interest and a protein known as Cas9, to make precise cuts in the genome. CRISPR gene editing enables scientists to easily locate and remove harmful genetic sequences and replace them with either neutral or beneficial genetic material (Doudna). Contrary to other gene-editing technologies, CRISPR is unique in its ability to create heritable traits that can be passed on through what is known as germline editing. In the context of human diseases, CRISPR provides a relatively simple approach to treat and even prevent debilitating genetic diseases such as Sickle Cell Anemia, Duchenne Muscular Dystrophy, and Cystic Fibrosis ().
The current regulatory regime for CRISPR gene editing is inapplicable and inefficient. Scientists using human embryos for CRISPR genome editing are incapable for qualifying for federal funding, and therefore, they either (1) abandon these projects entirely or (2) seek private funding. Privately funded projects allow researchers to bend ethical boundaries because they are not held to any standardized regulations (Tomlinson). This barrier in securing funding for CRISPR-based therapies stifles innovation because researchers are less likely to undertake related projects.
Currently, the Federal Drug Administration (FDA) treats CRISPR gene editing as a biological drug or device; however, these standards cannot be cross-applied to gene-editing technologies because they operate in entirely different ways. While the FDA’s approval is necessary for conventional medical treatments used on patients, simple laboratory research on embryos isn’t covered. For now, the FDA is not even allowed to review germline editing experiments in humans (Cohen). Consequently, researchers and companies using CRISPR are unaware of the laws that govern the therapies in which they have invested millions of dollars in until they are trying to gain market approval of these products.
While I understand the controversy surrounding embryo research, I think neglecting the reality and potential of CRISPR gene-editing in humans undermines the speed and credibility of biotechnological innovation. It is better to have a precautionary system in place that outlines the expectations of researchers in responsibly producing CRISPR-based therapies for gene-editing in human embryos rather than scramble to come up with one once the worst-case scenario ensues.
Moving forward, I propose that we immediately addresses the challenges in securing funding and gaining pre-market approval for CRISPR-based treatments on human embryos in order to develop life-saving therapies to treat genetic diseases. I would recommend that the National Institutes of Health be responsible for drafting guidelines on how to responsibly conduct research on human embryos in developing gene editing therapies and only fund those proposals that fulfill the respective criteria. This allows for standardization of gene-editing protocols and sets a baseline for what falls under the curtain of “ethical research.” Moreover, I would recommend that the FDA create a separate regulatory pathway to specifically evaluate gene-editing therapies.
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