Menu

Immunity May Make CRISPR-Based Therapies Ineffective

Researchers identify antibodies for two commonly used Cas9 proteins in human blood. Investors take notice.

Jan 10, 2018
Jim Daley

© BRYAN SATALINOCRISPR-Cas9 has been hailed as a breakthrough tool in genome editing that could usher in an era of precision gene therapy, and clinical trials are already scheduled to begin this year. But there may be a snag: our own immune systems.

The technique relies on Cas9 nucleases found in bacteria. But the majority of humans may have preexisting immune responses to bacteria from which the most common Cas9 homologs are derived, according to a preprint published last week (January 5) on bioRxiv.

The study, as MIT Technology Review first reported, examined the blood of 12 adults and 22 newborns for antibodies to Cas9 proteins from Staphylococcus aureus and Streptococcus pyogenes. They found antibodies for S. pyogenes in 65 percent of donors and antibodies for S. aureus in 75 percent—and nearly half of the donors had CD4+ T-cells that specifically targeted Cas9 homologs from S. aureus.

The immunity could not only limit the effectiveness of CRISPR, but also “create safety concerns,” the researchers write in the study. However, the problem is mostly limited to therapies that seek to edit genomes in vivo, they note—those that involve removing cells and editing genes in the lab may not be affected by this discovery.

“Like any new technology, you want to identify potential problems and engineer solutions for them,” study coauthor Matthew Porteus of Stanford University tells STAT News. “And I think that’s where we’re at. This is an issue that should be addressed.”

Executives at genome-editing companies, however, insist that the issues raised by this study “were either already being addressed or were not relevant to the medicines being developed,” according to STAT News

Shares of Intellia Therapeutics, Editas Medicine, and CRISPR Therapeutics were down the Monday after the paper was published, STAT News reports. 

January 2019

Cannabis on Board

Research suggests ill effects of cannabinoids in the womb

Marketplace

Sponsored Product Updates

WIN a VIAFLO 96/384 to supercharge your microplate pipetting!
WIN a VIAFLO 96/384 to supercharge your microplate pipetting!
INTEGRA Biosciences is offering labs the chance to win a VIAFLO 96/384 pipette. Designed to simplify plate replication, plate reformatting or reservoir-to-plate transfers, the VIAFLO 96/384 allows labs without the space or budget for an expensive pipetting robot to increase the speed and throughput of routine tasks.
FORMULATRIX® digital PCR technology to be acquired by QIAGEN
FORMULATRIX® digital PCR technology to be acquired by QIAGEN
FORMULATRIX has announced that their digital PCR assets, including the CONSTELLATION® series of instruments, is being acquired by QIAGEN N.V. (NYSE: QGEN, Frankfurt Stock Exchange: QIA) for up to $260 million ($125 million upfront payment and $135 million of milestones).  QIAGEN has announced plans for a global launch in 2020 of a new series of digital PCR platforms that utilize the advanced dPCR technology developed by FORMULATRIX combined with QIAGEN’s expertise in assay development and automation.
Application of CRISPR/Cas to the Generation of Genetically Engineered Mice
Application of CRISPR/Cas to the Generation of Genetically Engineered Mice
With this application note from Taconic, learn about the power that the CRISPR/Cas system has to revolutionize the field of custom mouse model generation!
Translational Models of Obesity, Dysmetabolism, Diabetes, and Complications
Translational Models of Obesity, Dysmetabolism, Diabetes, and Complications
This webinar, from Crown Bioscience, presents a unique continuum of translational dysmetabolic platforms that more closely mimic human disease. Learn about using next-generation rodent and spontaneously diabetic non-human primate models to accurately model human-relevant disease progression and complications related to obesity and diabetes here!