A genetic “Swiss Army knife” has been created that could treat incurable inherited diseases such as cystic fibrosis, sickle cell anemia and Parkinson’s.
The technology is an improvement on the innovative Crispr gene editing procedure first developed in 2012, which is possibly the most powerful medicinal tool ever created.
Crispr has enormous potential as a treatment, but one of the problems holding it back is that Cas9, the bacterial-derived enzyme that cuts and replaces defective or undesirable genes, is too large to be easily packaged, which limits its uses. .
Finding a smaller, but equally effective alternative has been a priority for several years.
A team of researchers at Stanford University focused on Cas14, a relative of Cas9 that had less than half the volume. However, it does not work naturally in mammals.
Dr. Lei Qi and her team tried to genetically manipulate the enzyme Cas14 to fix it and named its creation CasMINI.
“There were previous efforts by others to improve the performance of working CRISPRs. But our work is the first to make one work that doesn’t work,” Dr. Qi said.
“This is a critical step forward for CRISPR genome engineering applications,” added study lead author Stanley Qi.
“The work presents the smallest CRISPR to date, to our knowledge, as a genome editing technology. If sometimes people think of Cas9 as molecular scissors, here we have created a Swiss Army knife that contains multiple functions. It’s not big, but a very portable miniature to make it easier to use. ”
CasMINI’s tiny scale adds even more to the arsenal of Crispr technology, making it even easier to use and opening up more diseases to attack.
“The availability of a miniature CasMINI allows for new applications,” says Dr. Qi.
“On our wish list it will become a therapy to treat genetic diseases, cure cancer and reverse organ degeneration.”