Taylor Kubota said Bioengineers have repurposed a non-working CRISPR system to make a smaller version of the genome engineering tool. Its diminutive size should make it easy to deliver into human cells, tissues, and the body of gene therapy.
The simple analogy for CRISPR gene editing is that it operates like molecular scissors, cutting out select sections of DNA.
CRISPR can be as simple as a cutter, or more advanced as a regulator. Many applications are appearing from this impressive field, says Qi, assistant professor of chemical and systems biology in the Stanford School of Medicine and a Stanford Chem-H-institute scholar.
Different CRISPR systems in use or being clinically tested for gene therapy of diseases in the eye, liver, brain, remain limited in scope as they all suffer from the same defect. They are too large and too hard to deliver into cells, tissues, or living organisms.
Qi and his collaborators publish that they took a major step forward for CRISPR an efficient multi-purpose, mini CRISPR system. “CasMINI” has 529 amino acids.
The researchers confirmed in an experiment that CasMINI could delete, activate and edit genetic code.
The smaller the size it should be easier to deliver human cells into the human body and making a potential tool for treatment.
