Overcoming the AAV Size Limitation for CRISPR Delivery
Type
Blog Post
Published
Sept. 16, 2020, 1 p.m.
...aureus Cas9 (SaCas9, 1053 amino acids) displayed cleavage activity in mammalian cells. SaCas9 produced indels...vivo studies.
Testing AAV-SaCas9 in vivo
To test the efficiency of AAV-SaCas9 in vivo, the Zhang lab created...SpCas9, leading the group to focus their efforts on SaCas9 characterization for in vivo studies.
One of the...To compare the off-target effects of SpCas9 and SaCas9, Zhang’s group used an approach called BLESS (direct...). Using this sensitive method, they found that SaCas9 did not display higher levels of off-target activity...created an all-in-one SaCas9 and sgRNA construct using the liver-specific serotype AAV8. Since the efficiency...compared to a control AAV-GFP. Not only did the AAV-SaCas9-sgRNA constructs mediate genome modification, but...