Miniaturized CRISPR Packs a Mighty Gene Editing Punch
1 min read
Summary
Gene editing technology CRISPR-Cas9 has transformed the bio-tech sector, enabling scientists to gain insights into the function of genes, and how they can go wrong.
However, the technology’s size has limited its therapeutic applications, with it proving difficult to deliver the gene editor to cells in the muscle, brain, heart and other tissues, meaning its therapeutic applications have been limited to the liver and ex vivo treatments.
Now California-based Mammoth Biosciences, co-founded by CRISPR pioneer Jennifer Doudna, has developed NanoCas, which slashes the size of Cas9, the key component of CRISPR, to one-third of its original size.
This has allowed it to be packaged into a single delivery ‘box’ in the form of a virus that can be used for gene therapy inside the human body.
In tests on mice and monkeys, the team used NanoCas to edit genes involved in inherited high cholesterol and Duchenne muscular dystrophy.
The technology could be reworked for use in epigenetic or base editing, and the study suggests small Cas proteins can be mighty, broadening the scope of gene therapy.
