Key result
CASAAV platform enables temporally controlled, cell type-selective gene knockout in vivo within ~1 month.
Why the study?
In vivo loss-of-function studies are limited by the need for appropriate conditional knockout alleles and difficulty deploying CRISPR components in vivo.
Population
Cre-dependent Cas9-P2A-GFP mice focusing on cardiomyocytes
Comparison
CASAAV platform using AAV to deliver guide RNAs and Cre recombinase vs no gene disruption
Design
Preclinical study using CRISPR/Cas9/AAV-based somatic mutagenesis platform
Follow-up
Within a month
Authors
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May accelerate preclinical cardiomyocyte gene studies; leaves open human translation.
The CASAAV platform provides a rapid and efficient method for in vivo cell type-selective gene knockout in cardiomyocytes, overcoming limitations of traditional conditional knockout models.
VanDusen et al. (2017) studied this question. CASAAV platform (CRISPR/Cas9/AAV-based somatic mutagenesis) was evaluated on Temporally controlled, cell type-selective gene knockout. The CASAAV platform enables temporally controlled, cell type-selective gene knockout in vivo within a month using recombinant AAV to deliver guide RNAs and Cre recombinase to Cas9-P2A-GFP mice.
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