Overcoming the AAV Size Limitation for CRISPR Delivery
Type
Blog Post
Published
Sept. 16, 2020, 1 p.m.
...indel formation and phenotypic changes as early as 1 week post-injection. Livers from these mice were histologically...described above showed successful target modification, indicating that AAV is a good delivery vehicle for... with AAV. This system fuses catalytically dead dCas13b to the catalytic domain of RNA deaminase ADAR2...overall packaging capacity but necessitating purification and co-infection of two AAVs.
Cas9 orthologs...Streptococcus thermophilus and a rationally-designed truncated Cas9. Unfortunately, certain drawbacks limit the...that limits the number of targetable loci, and truncated Cas9 has much lower efficiency than its wild-type...the AAV-SaCas9-sgRNA constructs mediate genome modification, but they did so without a substantial immune...