COM Outlook Summer/Fall 2019

28 | DR. KIRAN C. PATEL COLLEGE OF OSTEOPATHIC MEDICINE Humans have directed genetic modifications for thou- sands of years through selective breeding. However, the precise mechanism was not truly understood until the discovery of DNA and the genetic code. We are at a cru- cial point today with a breakthrough called the CRISPR/ Cas9 system—short for Clustered Regularly Interspaced Short Palindromic Repeat DNA Sequences/CRISPR- associated protein 9 system, hereafter called CRISPR. CRISPR grabs our attention because it enables potent, specific, and accurate methods of genome editing in every type of cell (bacterial, animal, and plant). It is an extremely versatile and equally contro- versial new technology. Nova Southeastern University (NSU) is uniquely poised as an educational and clinical research hub, and the clinical faculty members can benefit from gaining expertise in new technologies with a wide range of medical applications. Nonetheless, as NSU faculty members venture into this new research arena, it is important to consider the ethical implications of CRISPR’s capabilities. Background The CRISPR system is an ancient bacterial immune system discovered in 1987. However, it was only when the Cas genes associated with DNA repeats were discovered in 2002 that a major door was opened in the field. In 2012, the idea for using CRISPR as a genome engineering tool was published by Jennifer Doudna, Ph.D., and Emmanuel Carpenter, Ph.D., which led to CRISPR being used in mouse and human cells by Feng Zhang, Ph.D., by 2013. The CRISPR technology is a two-part system composed of an endonuclease protein (Cas9) whose DNA-targeting specificity and cutting activity can be programmed by a short guide RNA (gRNA). The gRNA only allows Cas9 to function where there is an exact match for the sequence it carries. The gRNA carries a 20-letter sequence, which targets the region of the native genomic DNA. This 20-letter sequence is modular. This modular targeting system allows us to insert, delete, or modify DNA at a precise location on the genome, thus, making the GENETIC EDITING The Marvels and Concerns of a Revolutionary Technology FACULTY PERSPECTIVE FACULTY Perspective BY SAMIKSHA PRASAD, PH.D., AND VICKI TOSCANO, PH.D., J.D.