Gene-editing technology: potential to usher in a new age in medicine

“There’s going to be human gene editing therapies, discovery of new drugs, and new drug targets, CRISPR systems are being used in diagnostics including for SARS-COV-2, vector control, increased crop and food output,” said Neville Sanjana, a New York University professor who spoke virtually at the meeting on his team’s research.

Sanjana cited recently-published research relating to CRISPR with SARS-COV2, as an example of the promise that this technology holds. While the research into vaccines for COVID-19, has provided “great cause for optimism” in drastically reducing the impact that this pandemic has had on the population, vaccines do have their limitations, including not being broadly available across the world and the potential introduction of new viral variants. Using gene-editing technology, researchers may be able hone in on new therapeutic targets if they are needed, he said.

As an example, Sanjana said that his lab at the New York Genome Center and NYU in collaboration with Ben tenOever, PhD, at Mt. Sinai's School of Medicine used “top-ranked hits from the genome-scale CRISPR screen,” to identify key human genes requires for viral infection.1 Using human lung cells expressing the ACE2 receptor, they found that many of these genes were involved in the endosomal entry pathway, such as RAB7A. More importantly, inhibition of several top-ranked genes from the CRISPR screen led to a robust reduction (>100-fold) in SARS-CoV-2 infection of those human lung cells, demonstrating how CRISPR-identified genes can be used to discover new therapeutics.

Research like this is key to honing in on how the virus functions, and potential therapeutic targets, he noted.

Sanjana was part of the NYU and New York Genome Center research team that also recently developed chemically modified guide RNAs for a CRISPR system that targets RNA, intead of DNA, enhancing their ability to target, trace, edit, and or “knockdown,” RNA in human cells.

Questioned by an audience member on the cost involved in this type of research, Sanjana discussed NPR’s series on Victoria Gray, a woman who became the first patient in the United States to have her sickle cell disease treated using CRISPR technology. The series noted that the treatment was close to a million dollars. While he acknowledged the field is still very much in its infancy, and much more needs to be done to address the costs involved in manufacturing these types of health care answers, he feels as the technology continues to evolve, more research will enable more streamlined, and cost-effective ways to deliver these solutions.

“There is so much potential for this technology to help the world and we have to stay focused on that,” Sanjana said.

References

1. Daniloski Z, Jordan JK, Wessels HH, et al. Identification of required host factors for SARS-COV-2 infection in human cells. Cell.2021; 184: 92-105.

2. Sanjana N. Promises and challenges of gene editing in the age of CRISPR. Presented at The North American Menopause Society Annual Meeting. September 24, 2021.