Screening and validation of genome-edited animals

2021 ◽  
pp. 002367722110169
Author(s):  
Rosie K Bunton-Stasyshyn ◽  
Gemma F Codner ◽  
Lydia Teboul

The emergence of an array of genome-editing tools in recent years has facilitated the introduction of genetic modifications directly into the embryo, increasing the ease, efficiency and catalogue of alleles accessible to researchers across a range of species. Bypassing the requirement for a selection cassette and resulting in a broad range of outcomes besides the desired allele, genome editing has altered the allele validation process both temporally and technically. Whereas traditional gene targeting relies upon selection and allows allele validation at the embryonic stem cell modification stage, screening for the presence of the intended allele now occurs in the (frequently mosaic) founder animals. Final confirmation of the edited allele can only take place at the subsequent G1 generation and the validation strategy must differentiate the desired allele from a range of unintended outcomes. Here we present some of the challenges posed by gene editing, strategies for validation and considerations for animal colony management.

2020 ◽  
Vol 45 ◽  
pp. 101837 ◽  
Author(s):  
Vera Alexeeva ◽  
Iraz T. Aydin ◽  
Christoph Schaniel ◽  
Alec W. Stranahan ◽  
Sunita L. D'Souza ◽  
...  

Cancers ◽  
2021 ◽  
Vol 13 (13) ◽  
pp. 3182
Author(s):  
Lorena López-Ferreras ◽  
Nicole Martínez-García ◽  
Laura Maeso-Alonso ◽  
Marta Martín-López ◽  
Ángela Díez-Matilla ◽  
...  

The p53 family has been widely studied for its role in various physiological and pathological processes. Imbalance of p53 family proteins may contribute to developmental abnormalities and pathologies in humans. This family exerts its functions through a profusion of isoforms that are generated by different promoter usage and alternative splicing in a cell type dependent manner. In particular, the Trp73 gene gives rise to TA and DN-p73 isoforms that confer p73 a dual nature. The biological relevance of p73 does not only rely on its tumor suppression effects, but on its pivotal role in several developmental processes. Therefore, the generation of cellular models that allow the study of the individual isoforms in a physiological context is of great biomedical relevance. We generated specific TA and DN-p73-deficient mouse embryonic stem cell lines using the CRISPR/Cas9 gene editing system and validated them as physiological bona fide p73-isoform knockout models. Global gene expression analysis revealed isoform-specific alterations of distinctive transcriptional networks. Elimination of TA or DN-p73 is compatible with pluripotency but prompts naïve pluripotent stem cell transition into the primed state, compromising adequate lineage differentiation, thus suggesting that differential expression of p73 isoforms acts as a rheostat during early cell fate determination.


2017 ◽  
Vol 29 (1) ◽  
pp. 1 ◽  
Author(s):  
Santiago Josa ◽  
Davide Seruggia ◽  
Almudena Fernández ◽  
Lluis Montoliu

Gene editing is a relatively recent concept in the molecular biology field. Traditional genetic modifications in animals relied on a classical toolbox that, aside from some technical improvements and additions, remained unchanged for many years. Classical methods involved direct delivery of DNA sequences into embryos or the use of embryonic stem cells for those few species (mice and rats) where it was possible to establish them. For livestock, the advent of somatic cell nuclear transfer platforms provided alternative, but technically challenging, approaches for the genetic alteration of loci at will. However, the entire landscape changed with the appearance of different classes of genome editors, from initial zinc finger nucleases, to transcription activator-like effector nucleases and, most recently, with the development of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas). Gene editing is currently achieved by CRISPR–Cas-mediated methods, and this technological advancement has boosted our capacity to generate almost any genetically altered animal that can be envisaged.


2018 ◽  
Vol 27 ◽  
pp. 131-135 ◽  
Author(s):  
Ruoji Zhou ◽  
An Xu ◽  
Donghui Wang ◽  
Dandan Zhu ◽  
Helen Mata ◽  
...  

2019 ◽  
Vol 12 (1) ◽  
Author(s):  
Dingyu Liu ◽  
Can Huang ◽  
Jiaxin Guo ◽  
Peiji Zhang ◽  
Tao Chen ◽  
...  

Abstract Background Metabolic engineering has expanded from a focus on designs requiring a small number of genetic modifications to increasingly complex designs driven by advances in multiplex genome editing technologies. However, simultaneously modulating multiple genes on the chromosome remains challenging in Bacillus subtilis. Thus, developing an efficient and convenient method for B. subtilis multiplex genome editing is imperative. Results Here, we developed a CRISPR/Cas9n-based multiplex genome editing system for iterative genome editing in B. subtilis. This system enabled us to introduce various types of genomic modifications with more satisfying efficiency than using CRISPR/Cas9, especially in multiplex gene editing. Our system achieved at least 80% efficiency for 1–8 kb gene deletions, at least 90% efficiency for 1–2 kb gene insertions, near 100% efficiency for site-directed mutagenesis, 23.6% efficiency for large DNA fragment deletion and near 50% efficiency for three simultaneous point mutations. The efficiency for multiplex gene editing was further improved by regulating the nick repair mechanism mediated by ligD gene, which finally led to roughly 65% efficiency for introducing three point mutations on the chromosome. To demonstrate its potential, we applied our system to simultaneously fine-tune three genes in the riboflavin operon and significantly improved the production of riboflavin in a single cycle. Conclusions We present not only the iterative CRISPR/Cas9n system for B. subtilis but also the highest efficiency for simultaneous modulation of multiple genes on the chromosome in B. subtilis reported to date. We anticipate this CRISPR/Cas9n mediated system to greatly enhance the optimization of diverse biological systems via metabolic engineering and synthetic biology.


2011 ◽  
Author(s):  
Moon Nian Lim ◽  
Umapathy Thiageswari ◽  
Othman Ainoon ◽  
P. J. N. Baharuddin ◽  
R. A. Jamal ◽  
...  

Sign in / Sign up

Export Citation Format

Share Document