A step toward a more efficient way to make gene therapies to attack cancer, genetic disorders

A UCLA-led research team today reports that it has developed a new method for delivering DNA into stem cells and immune cells safely, rapidly and economically. The method, described in the journal Proceedings of the National Academy of Sciences, could give scientists a new tool for manufacturing gene therapies for people with cancer, genetic disorders and blood diseases.

The study’s co-senior author is Paul Weiss, a UCLA distinguished professor of chemistry and biochemistry, of bioengineering and of materials science and engineering. “We are figuring out how to get gene-editing tools into cells efficiently, safely and economically,” he said. “We want to get them into enormous numbers of cells without using viruses, electroshock treatments or chemicals that will rip open the membrane and kill many of the cells, and our results so far are promising.”

Dr. Steven Jonas, the study’s co-senior author, a UCLA clinical instructor in pediatrics and member of the California NanoSystems Institute at UCLA, the UCLA Jonsson Comprehensive Cancer Center and Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and former UCLA CTSI KL2 awardee, said the research has the potential to benefit adults and children with cancer, immune system disorders and genetic diseases. 

UCLA CTSI and UC CAI congratulate Drs. Weiss and Jonas on their study. They received a University of California Center for Accelerated Innovation (UC CAI) Stimulus Award in 2017 to support this research and later received an UC CAI Technology Development Grant Award. As a UCLA CTSI KL2 awardee, Jonas received support from the KL2 program to obtain the NIH Director’s Early Independence Award--funding which also contributed to this research.

The study was also funded in part through Belling’s predoctoral fellowship through the National Heart, Lung, and Blood Institute. Jonas also has received young investigator awards from the Alex’s Lemonade Stand Foundation for Childhood Cancer Research, Hyundai Hope on Wheels Foundation for Pediatric Cancer Research, and the Tower Cancer Research Foundation. UCLA’s Technology Development Group Innovation Fund also provided funding.

The study’s lead author is Jason Belling, a UCLA graduate student in chemistry and biochemistry. Other co-authors include Duke University professor Tony Huang, a pioneer of acoustofluidics and a UCLA alumnus; Dr. Stephen Young, distinguished professor of medicine and human genetics at the David Geffen School of Medicine at UCLA; and Dr. Satiro De Oliveira, a UCLA assistant professor of pediatrics.

Read the full UCLA press release. 

Image source: Reed Hutchinson/UCLA
Image caption: Dr. Steven Jonas, Jason Belling and Paul Weiss.