Innovations and Opportunities in Genome Editing

CRISPR/Cas9 has revolutionized the gene editing platform. Frost & Sullivan has closely followed its evolution and its implications on health care and other industries for several years. Read more about how the future of pharmaceuticals, therapy, and health care in general may rest to a large extent on gene editing.

The possibilities and the fantasy associated with genetic technology have captured the public’s imagination like no other technological development. Associated stakeholders have been extremely supportive of the technology for decades, despite abortive promises. Frost & Sullivan has closely followed its evolution and its implications on health care and other industries for several years. There are compelling reasons to believe that the future of pharmaceuticals, therapy, and health care in general will rest to a large extent on gene editing.

Foremost among these is the evolution of the gene editing tool CRISPR/Cas9. CRISPR is an abbreviation of clustered regularly interspaced short palindromic repeats, a family of DNA sequences that naturally occur in bacteria and other microorganisms. The set of genes located next to the CRISPR sequences are referred to as a CRISPR-associated system (Cas). Cas9 refers to a specific protein that makes targeted snipping possible. RNA molecules guide Cas9 proteins toward the exact location on the DNA that has to be edited. Together, the CRSIPR/Cas system confers the host with immunity against foreign genetic agents, and it is possible to induce heightened immune resistance to an organism by editing its genome with the CRISPR/Cas9 tool. A sampling of gene editing innovations and their diverse applications is provided below to illustrate the versatility and power of this tool.

CRISPR Platform for Hearing Loss (Howard Hughes Medical Institute, Chevy Chase, Md.)

Principal investigator David Liu and his team are exploring possible genetic therapies to restore hearing loss caused by genetic conditions—one of which is a mutation of the gene Tmc1 that causes the loss of inner ear cells that sprout fine hair strands. It is these bristles that detect sound waves, and the pattern of change is recognized as meaningful sound. The scientists use the CRISPR/Cas9 tool to snip out the specific mutated Tmc1 gene. In animal models, it was observed that the injection of a cocktail of gene editing tools alleviated or slowed the progress of hearing loss. The findings of this study were published in the journal Nature in December 2017.

Improving Algal Yield through Gene Editing (University of Edinburgh, Scotland)

Climate change, water shortages, and the spread of pests and infectious diseases have been adding pressure to agricultural practices. These factors not only make farming more expensive and unpredictable, but also significantly affect yields. Scientists have long studied ways to improve plants’ disease resistance, and one approach that is showing great promise is gene editing. Scientists at the University of Edinburgh have developed an innovative method that improves algae yields. The researchers have modified the genes of a green alga, Chlamydomonas reinhardtii, by using CRISPR molecules. This approach adds specific DNA segments to the algal genome in order to make it more resilient to harsh climates and diseases, and it was observed to improve the yield. The study demonstrating the efficiency of this technique was published in the Proceedings of the National Academy of Sciences in November 2017, and speaks to the scalability of the technique, which the scientists believe will have a big impact on the industries that depend on algal products: biofuels, agriculture, cosmetics, and biotechnology.

Clipping Mosquitoes’ Wings through Genetic Modifications (University of California, Riverside)

As fun as camping can be, one cannot escape mosquitoes—more specifically, the annoying buzzing sound they make as fly by. The creation of wingless mosquitoes and flies would not merely make the lives of recreational campers better, but prevent the spread of insect-borne pathogens. A number of deadly diseases—malaria, dengue and yellow fever, to name a few—are spread by mosquitoes. The dream of creating flightless mosquitoes has lingered in the scientific community for decades; however, ineffective genetic tools and the poor survival of the mutated insects have repeatedly thwarted such efforts.  UC Riverside scientists have created transgenic, wingless mosquitoes, using CRISPR to disrupt the genes that control the insects’ vision and flight.  The careful introduction of genetically modified insects into the general population so they can mate and produce offspring with inherited mutations could significantly reduce that population—and their potential to spread disease—in just a few breeding cycles.

The Road Ahead

CRISPR/Cas9 has revolutionized the gene editing platform. Its simplicity and ease of use have prompted many academic and industry researchers to adopt the technology for gene editing. From agriculture to pharmaceuticals, biotechnology to environmental science, a gamut of industrial applications are riding on the power of gene editing. Biotechnology seems to be the low-hanging fruit, and the industry that is most likely to benefit from gene editing in the next few years. The public’s concerns about genetic engineering of any kind must be allayed for some of these innovations to make a significant difference.

Copyright © 2018 Frost & Sullivan