Multistep allelic conversion in mouse pre-implantation embryos by AAV vectors

Author:

Nickl Petr1,Jenickova Irena2,Elias Jan1,Kasparek Petr1,Kopkanova Jana1,Barinka Cyril3,Sedlacek Radislav1

Affiliation:

1. Institute of Molecular Genetics of the Czech Academy of Sciences

2. Czech Centre for Phenogenomics, Institute of Molecular Genetics of CAS

3. Institute of Biotechnology of the Czech Academy of Sciences, BIOCEV

Abstract

Abstract

Site-specific recombinases (SSRs) are critical for achieving precise spatiotemporal control of engineered alleles. These enzymes play a key role in facilitating the deletion or inversion of loci flanked by recombination sites, resulting in the activation or repression of endogenous genes, selection markers or reporter elements. However, multiple recombination in complex alleles can be laborious. To improve this, a new and efficient method using AAV vectors can simplify the conversion of systems based on Cre, Flpo, Dre and Vika recombinases. In this study, we present an effective method for ex vivo allele conversion using Cre, Flp (flippase), Dre, and Vika recombinases, employing adeno-associated viruses (AAV) as a delivery vector. AAVs enable efficient allele conversion with minimal toxicity in a reporter mouse line. Moreover, AAVs facilitate sequential allele conversion, essential for fully converting alleles with multiple recombination sites, typically found in conditional knockout mouse models. While simple allele conversions show a 100% efficiency rate, complex multiple conversions consistently achieve an 80% conversion rate. Overall, this strategy markedly reduces the need for animals and significantly speeds up the process of allele conversions, representing a significant improvement in genome engineering techniques.

Publisher

Springer Science and Business Media LLC

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