Non-CRISPR Gene Editing Platforms Make the Cut--or Avoid It | GEN

This article discusses research by �黨�ǿ���Ƶ Senior Scientist Joshua Rosenthal, co-founder of biotech company Korro Bio.

Easy-to-use nuclease platforms have democratized , especially in the laboratory setting. But these platforms, as they exist today, are less precise than alternative genome editing systems, such as zinc finger nuclease (ZFN) systems and transcription activator-like effector nuclease (TALEN) systems. Indeed, the use of CRISPR platforms can result in off-target modifications, unwanted on-target modifications, and genomic rearrangements. Consequently, CRISPR platforms are being developed that will be more precise.

Even though CRISPR platforms are bound to improve, we shouldn’t be content to put all our genome editing eggs in one basket. We should be aware that the genome editing systems that preceded CRISPR—ZFN systems and TALEN systems, for example—are anything but static. They’ve been improving alongside CRISPR. Also, we should be aware of the newer systems that have been following CRISPR’s example.

Cephalopods have very high levels of plasticity in protein expression, mostly in nervous system cells. One of the ways they sustain this plasticity is through the use of Adenosine Deaminase Acting on RNA (ADAR) enzymes. Josh Rosenthal, PhD, a neurobiologist at the Marine Biological Laboratory and a co-founder of Korro Bio, discovered that ADAR could make an edit in mRNA using a chemical moiety.