Single-AAV Delivery of the SpCas9 Nickase Improves CAG/CTG Repeat contraction in a mouse model of Huntington's disease
The best studied Cas9 enzyme, from Streptococcus pyogenes, is thought to be too large to be efficiently packaged into a single adeno-associated virus (AAV) together with a single-guide (sg) RNA cassette, requiring a dual-vector system. Here we have circumvented this limitation and packaged the full CRISPR system into a single AAV by using a single promoter driving the expression of both the Cas9…
We haven't written up this one. bioRxiv has the full story — the link below goes straight to it.
