As CRISPR human trials are just getting off the ground (see Genome printing: what is the status of human treatment projects?), the NIH[1] is set to finance a study assessing the safety of genome printing in human cells and tissues. The team, led by Professor Todd McDevitt at the Gladstone Institute, will therefore receive 3.6 million dollars over five years. He assembled “a team of leading investigators with complementary experience in tissue engineering, genome editing, gene therapy, stem cell biology and single-cell genomics” to “develop platforms that can accurately detect adverse effects of genome editing on physiological functions”. “By evaluating possible safety and toxicity issues in human cells and tissues, we hope to help avoid unanticipated effects in future clinical trials”, announced McDevitt. Until now, the researchers have attempted to establish whether CRISPR-mediated genome changes affected DNA areas other than the target gene. The purpose of this new study is to establish whether genome editing causes unexpected changes in terms of cell or tissue function. The scientists want to focus primarily on cardiac and hepatic tissue as well as the brain.
Jennifer Doudna, Ph.D., researcher at Gladstone and Professor at UC Berkeley (see CRISPR: are Jennifer Doudna and Emmanuelle Charpentier responsible for the ethical scope of their discovery?) also received NIH funding to “expand the current tool kit for human genome editing by exploring new CRISPR-Cas proteins and enzymes that can repair DNA”.
[1] National Institute of Health.
Eurekalert (10/10/2018)