Faulty gene? CRISPR will repair it

CRISPR, genome editing technology, allows the cutting of a bit of mutated DNA and replacing it with a healthy strand. It is at the forefront of the vision of personalized medicine. In recent years, Israeli academia has been gaining expertise in this technology. Will excellence in fundamental research translate into future success for companies?

Dr. Ayal Hendel

In this era, the high-tech industry is producing innovative developments at an accelerated pace. Public discourse is mainly concentrated on autonomous vehicles, AI, and advanced robotics. Under the radar, however, a tremendous revolution is materializing in healthcare – the revolution of genome editing, which is sourced in biological research. The technology of genome editing is employed to alter and repair genes in order to cure diseases. The hopes of many are riding on this technology, because it offers direct access to the source of the defect to alter the genetic material – the DNA – in order to repair it. Genome editing technology corresponds well with personalized medicine, where doctors tailor therapies to patients’ individual genetic makeup in order to maximize efficacy and to reduce the risk of side effects.

Genome editing technology: The CRISPR system

Genome editing technology is based on the use of the CRISPR system, which allows the cutting away of a bit of mutated DNA and replacing it with a healthy DNA strand. The system is based on a mechanism discovered in bacteria, which protects them from attack by viruses by cutting the viruses’ DNA. In 2012, the journal Science published a groundbreaking article written by two researchers, Prof. Jennifer Doudna and Prof. Emmanuelle Charpentier, that illustrated the manner in which the CRISPR system can be engineered to edit any sequence of DNA. One year later, in the same journal, researcher Prof. Feng Zhang demonstrated how the system can be employed to edit DNA in human cells. While the technology is still young, the CRISPR system is now considered to be one of the most important and promising technologies in the pharmaceutical industry. It is already being employed in clinical trials of patients in the US, Europe and China. Further developments in genome editing technology could potentially lead to effective, precise, and safe cures for many diseases.

In recent years, Israeli academia has been attracting young scientists who specialize in genome editing from leading universities such as Stanford, Berkeley and MIT, to continue the development of this technology in Israeli laboratories.

For example, the laboratory for gene repair at Bar-Ilan University headed by Dr. Ayal Hendel, in collaboration with pediatrician Prof. Raz Somech’s research group from the Sheba Medical Center at Tel Hashomer, is working on the development of genome editing technology for bubble boy disease. Bubble boy disease (SCID – Severe Combined Immunodeficiency) is a general name for a group of genetic disorders marked by disturbed creation and function of immune cells. Children suffering from this disease can only live in sterile conditions; if left untreated, they do not survive past their first year. The most common treatment available today is a bone marrow transplant, but it is hard to find a matching donor, and a transplant from a donor who is not a full match could prove dangerous. The employment of genome editing to directly repair the faulty gene in the patient’s own cells could offer an alternative to bone marrow transplants. This method is the laboratory’s focal point of research.

This research is currently being complemented by the work of a handful of startups, but it is also the basis for the emergence of additional companies. This trend is providing Israel with a tremendous opportunity to be included in the global race for the advancement of genome editing technology as a tool for gene therapy and for the advancement of personalized medicine as a whole.

Written by Dr. Ayal Hendel, senior lecturer and principal investigator at the gene therapy laboratory in the Institute for Nanotechnology and the Faculty of Life Sciences at Bar-Ilan University