In the new study, published in January in the journal “Nature Biotechnology”, scientists from the University of California in Berkeley, under the leadership of Jennifer Dudna, co-author of the CRISPR-CAS9 genomic editing system, have introduced a new delivery method for CRISPR-CAS9. This method allows genetic editing directly in the body, which is a step towards creating software delivery methods that save patients from the need to destroy the bone marrow and the immune system before the introduction of edited blood cells.
CRISPR is a revolutionary technology in the world of genetics that opens up new horizons in the treatment of diseases and the study of living organisms. It is a genomic editing system that allows scientists to make accurate changes to the DNA. It is like having molecular “scissors” that can carefully cut and replace areas of the genetic code. This technology goes beyond treating the symptoms of diseases by providing the ability to eliminate the causes at the genetic level. CRISPR promises a breakthrough in the treatment of cancer, genetic diseases, and even in the fight against age-related changes. It is like the key to the secret room of nature, where the secrets of life are hidden, and now scientists have a tool for their disclosure.
The new approach utilizes shells obtained from viruses to create “shell vehicles” that can deliver CAS9 proteins, RNA-directors, and transgens directly to specific types of human T cells. These cells are then transformed into cancer fighters. In a demonstration, researchers transformed human T cells in mice with a humanized immune system into CAR T-cells that are capable of attacking and destroying other classes of immune cells.
This method is one of several experimental approaches for the delivery of gene therapy. The study successfully proves that scientists are not only able to deliver a transgen for a receptor targeted at cancer cells, but also exclude receptors that are not specifically aimed at cancer using CRISPR.
Jennifer Dudna and her laboratory are continuing to improve the efficiency of delivery using EDV. The main goal is to make CRISPR therapy more affordable and cost-effective. Dudna emphasizes that new technologies enabling genetic editing directly in the body will be instrumental in reducing prices for such therapy and overcoming inequality in access to expensive gene therapies.