Gene Editing Research Could Yield Effective New Treatments

January 28, 2020

The University of Texas Health Science Center at Tyler has recently begun research in gene editing aimed at discovering effective new treatments for an array of diseases and infections. Joining UT Health Science Center at Tyler in late 2019, Guohua Yi, PhD, brought along a three-year $2.25 million National Institutes of Health (NIH)-funded grant to explore the potential viability of gene editing, or genome engineering, to treat genetic and infectious diseases.

Simply put, gene editing is a group of technologies that provide scientists the ability to change an organism’s DNA. These technologies allow genetic material to be added, removed or altered at particular locations within the genome. While several approaches to genome engineering have been developed, Dr. Yi is specifically studying the successful and effective delivery of clustered regularly interspaced short palindromic repeats and associated protein 9 (CRISPR-Cas9) to T cells.

CRISPR-Cas9 allows the alteration of a cell’s DNA at precise locations by utilizing a specially designed RNA molecule to guide a Cas9 enzyme to a specific sequence of DNA. Within this specific stretch of genetic code, Cas9 then cuts the strands of DNA at that point and removes a small segment, creating a gap in the structure where a new piece can be added. This process can modify genes in living cells and organisms, which may yield the possibility to correct mutations at exact locations in the human genome, effectively treating disease. In this case, Dr. Yi is focused on the effective delivery to a particular type of white blood cell, T cells. Also referred to as T lymphocyte and thymocyte, T cells are part of the immune system and play a vital role protecting the body from infection and disease.

CRISPR-Cas9, revered as a breakthrough in science, is quickly proving to be the frontrunner of gene editing and has generated much excitement in the scientific community due to its efficiency, accuracy, cost and speed. It has emerged as a powerful tool for genome engineering in diverse organisms, as well as for developing therapeutics for genetic and infectious diseases. As T cells play a critical role in orchestrating cell-mediated immunity and humoral immunity, T cell gene editing offers promise to treat such diseases as HIV and cancer, as well as autoimmune diseases. Moreover, the process may help to understand how diseases form and respond to treatment, as well as new methods for diagnosis, treatment and prevention.

However, delivery of CRISPR-Cas9 to T cells for efficient modification remains difficult. Therefore, Dr. Yi has set his sights on effectively creating a novel lentivirus-like particle linked to an antibody specific to T cells. This antibody-conjugated lentivirus will specifically bind to T cells and deliver the encapsulated CRISPR-Cas9 to T cells for successful gene editing.