CRISPR Genomic Editing: a Curse or a Boon?

Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) and Cas9 have together stolen the spotlight as arguably one of the greatest scientific breakthroughs in the past decade for synthetic biology. For years, gene therapy has been tabooed and labeled as a method that’s indiscriminate to the inherent specificity of the human genome. In 2000, a landmark clinical trial for severe combined immunodeficiency (SCID) hit the nail on the coffin for gene therapy when a seemingly cured patient was suddenly diagnosed with leukemia many months after the procedure. The three-year old patient was previously administered the appropriate corrective genes to replace the defective sequences, but the genes apparently were not integrated into a correct spot and tampered with genes necessary to bar uncontrolled cell division and cancer.

"Despite the bad reputation encircling CRISPR, its potential medicinal uses cannot be underestimated."

CRISPR and the ninth variant of the CRISPR associated protein complex (Cas9) provide the perfect solution to the problem of accuracy in the SCID case. Cas9, a nuclease, with the aid of a guide RNA, simply cuts out the targeted sequence allowing synthetic biologists to fill in the gap with the accurate sequence, all presumably without any adverse effects. However, now that we do have the tools to bring previous attempts at genomic editing to fruition, questions are arising regarding whether we forge ahead and implement CRISPR. Does CRISPR really shape up to be the dangerous double-edged sword it’s widely publicized to be?

One of the most serious questions surrounding CRISPR is the possible use in having “designer babies”. Even though the idea of CRISPR/Cas9 was conceived in a strictly medicinal context, the approach could be easily perverted to engineer specific traits into a child, including gender. Such genomic editing would immorally permit parents to pick and choose characteristics that they deem favorable, including specific traits that may bolster athletic or academic aptitude. Even if a future regulation were to guarantee its strictly medicinal uses, CRISPR/Cas9 hasn’t been perfected as of yet. According to Nature magazine, HIV has been shown to be able to surmount the effects of CRISPR genomic editing within just a few hours. In addition, to apply to already living people, we would have to first perfect gene transfer via viral vectors in order to properly administer the corrective genes, a step that has been stalled ever since its use in the SCID trials. Even with that taken for granted, according to the American Association for the Advancement of Science, a variety of diseases, including cystic fibrosis or muscular dystrophy, cannot be solved by simply transplanting corrective cells.

Despite the bad reputation encircling CRISPR, its potential medicinal uses cannot be underestimated. The idea of being able to systematically and accurately target any gene can serve as robust platforms for treatment of diseases like SCID or even Alzheimer's. Furthermore, to compensate for the idea of completely editing out a gene, a form of deactivated Cas9 (dCas9) has been modified to account for a system that only amplifies or slightly represses the expression of a specific gene. Some of these such renowned complexes (dCas9-KRAB and dCas9-VP) can fine-tune gene expression in certain cases such as diabetes mellitus, where the protein insulin has to be secreted at a specific level. In addition, once perfected, the activation/repression complexes could circumvent the murky ethics surrounding thorough editing. CRISPR/Cas9 approaches also entail a much more affordable treatment cost compared to some of the exorbitant finances needed for the current therapeutics in place. Combined with the fascinatingly wide gamut of diseases that it can cover (basically any that have been linked to specific genes), CRISPR genomic editing provides for a very cost-effective treatment. In addition, there’s no need to limit goals for using CRISPR techniques on human genomes. CRISPR/Cas9 can revolutionize the field of transgenic animals and genetically modified organisms (GMOs). If anything, CRISPR may be the answer to a variety of agricultural quandaries as well and can be moderately used to make beneficial contributions to the field of genetic modification.

In summary, CRISPR genomic editing is a very polarized topic in gene therapy. The sheer potential CRISPR has truly compelled us to quicken the pace of our clinical trials and approve the approach, but the tool can be evidently used for unspeakably immoral purposes. Though, CRISPR/Cas9 approaches merit supplementary research involving accurate gene transfer and nevertheless should not be taken off the table. If one thing is for sure, the advancement of CRISPR/Cas9-mediated approaches will not be constricted by the imagination of researchers or the robustness of the relevant techniques that are developed, but by the regulations that will hopefully stand to prevent immoral use.