Scientists at the University of California San Francisco have found a way to edit genomes, a method they say could revolutionize treatments for cancer, infections like HIV and autoimmune conditions like rheumatoid arthritis and lupus.
The research, published Wednesday in the journal Nature, is a “turning point,” Vincenzo Cerundolo, director of the Human Immunology Unit at Oxford University, told The Washington Post.
“It is a game-changer in the field, and I’m sure that this technology has legs.”
Scientists used CRISPR gene-editing technology to precisely remove genes from white blood cells of the immune system and insert beneficial replacements. The process utilizes electrical fields instead of viruses to insert DNA to make the cells permeable, speeding up the process significantly.
Graduate student Theo Roth “tested thousands of conditions” over the course of a year to figure out DNA quantity and which gene-editing tools and electrical fields to use to modify T-cells without a virus.
“Theo was convinced that if we could figure out the right conditions, we could overcome these perceived limitations, and he put in a Herculean effort to test thousands of different conditions: the ratio of the CRISPR to the DNA; different ways of culturing the cells; different electrical currents,” said UCSF’s Alex Marson, scientific director of biomedicine at the Innovative Genomics Institute and senior author of the new study. “By optimizing each of these parameters and putting the best conditions together, he was able to see this astounding result.”
The authors tested the method in two scenarios, including using CRISPR to fix a single mutation in T-cells from children with a rare genetic form of autoimmunity. The process took one to two weeks, and the family is now waiting on the FDA for approval to be treated with the repaired T-cells.
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