BRF Research News

Our goal for 2017 was to improve the efficiency of a new technology, “gene editing” by CRISPR, that can precisely edit genes in eggs activated to become stem cells. BRF scientists accomplished this goal in a mouse model by developing new methods that improve the efficiency of CRISPR gene editing in mouse eggs from 10% to approximately 75%, with the added success of deriving stem cells from more than 50% of the gene edited, activated eggs.

Two genes were simultaneously targeted for editing:

(1) Just as Type “O” blood can be given to almost everyone, a “universal” stem cell could be missing the gene, B2M, responsible for the proteins on stem cells that cause immune rejection following transplantation. Such a “universal” stem cell could be transplanted into many individuals without leading to immune rejection. This is an essential step to the derivation of “off-theshelf” stem cells for everybody.

The 2017 mouse egg stem cell experiments by BRF scientists derived mouse stem cells missing B2M. This paves the way to translate the research to the derivation of universal stem cells from human eggs. Like blood banks, universal stem cell banks would be available in hospitals for acute treatments, such as heart attack, stroke and spinal cord injury.

(2) CRISPR gene editing can also mimic the natural mutation in 1% of humans that renders individuals resistant to infection by HIV, the virus that causes AIDS. The recent success in mouse eggs to eliminate the HIV receptor, CCR5, paves the way to deriving a library of universal human stem cells also resistant to HIV infection.

IF those cells can be developed into bone marrow stem cells, and IF those bone marrow stem cells will function normally, they could be utilized as a powerful treatment, perhaps a cure, for HIV disease.