Precision BioSciences Inc (DTIL)

[NY1972]

DTIL mgt. should cut burn to allow in vivo programs to catch up.

Many DNA-editing enzymes have been used to shift heteroplasmy, but they have their pitfalls in terms of potential clinical use. MitoZFN’s and mitoTALENs have a heterodimeric architecture, making packaging into viral vectors difficult, requiring that each monomer is packaged into separate viral vectors. The CRISPR-Cas9 system is not appropriate for mtDNA modification, because mitochondria do not have an RNA import mechanism33. More recently, a base editor DdCBE was shown to edit cytosines preceded by thymidines34. However, the sequence requirements limit its potential use at this time35. It has, however, recently been used to edit mtDNA in mouse embryos36. MitoARCUS overcomes disadvantages that mitoZFN and mitoTALENs present with size and viral packaging.