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Updated: Oct 16, 2026

Loss- and Gain-of-function Approach to Investigate Early Cell Fate Determinants in Preimplantation Mouse Embryos
Published on: June 6, 2016
Targeted disruption of the cell-cycle checkpoint gene ATR leads to early embryonic lethality in mice
A de Klein1, M Muijtjens, R van Os
1MGC Department of Cell Biology and Genetics, Center for Biomedical Genetics, Erasmus University, Rotterdam, The Netherlands. deklein@gen.fgg.eur.nl
Abstract:
Checkpoints of DNA integrity are conserved throughout evolution, as are the kinases ATM (Ataxia Telangiectasia mutated) and ATR (Ataxia- and Rad-related), which are related to phosphatidylinositol (PI) 3-kinase [1] [2] [3]. The ATM gene is not essential, but mutations lead to ataxia telangiectasia (AT), a pleiotropic disorder characterised by radiation sensitivity and cellular checkpoint defects in response to ionising radiation [4] [5] [6]. The ATR gene has not been associated with human syndromes and, structurally, is more closely related to the canonical yeast checkpoint genes rad3(Sp) and MEC1(Sc) [7] [8]. ATR has been implicated in the response to ultraviolet (UV) radiation and blocks to DNA synthesis [8] [9] [10] [11], and may phosphorylate p53 [12] [13], suggesting that ATM and ATR may have similar and, perhaps, complementary roles in cell-cycle control after DNA damage. Here, we report that targeted inactivation of ATR in mice by disruption of the kinase domain leads to early embryonic lethality before embryonic day 8.5 (E8.5). Heterozygous mice were fertile and had no aberrant phenotype, despite a lower ATR mRNA level. No increase was observed in the sensitivity of ATR(+/-) embryonic stem (ES) cells to a variety of DNA-damaging agents. Attempts to target the remaining wild-type ATR allele in heterozygous ATR(+/-) ES cells failed, supporting the idea that loss of both alleles of the ATR gene, even at the ES-cell level, is lethal. Thus, in contrast to the closely related checkpoint gene ATM, ATR has an essential function in early mammalian development.
Insights
The Ataxia- and Rad-related (ATR) gene is essential for early mammalian development, as its complete inactivation leads to embryonic lethality. Unlike ATM, ATR plays a critical role in cell-cycle control and embryonic survival.
Area of Science:
- Molecular Biology
- Genetics
- Developmental Biology
Background:
- DNA integrity checkpoints are crucial for cell survival and are regulated by kinases like ATM and ATR.
- ATM mutations cause ataxia telangiectasia, while ATR's role in mammalian development was unclear.
- ATR is structurally related to yeast checkpoint genes and implicated in DNA damage response.
Purpose of the Study:
- To investigate the essentiality of the ATR gene in mammalian development.
- To determine the in vivo function of ATR by targeted gene inactivation in mice.
- To compare the roles of ATR and ATM in cell-cycle control and development.
Main Methods:
- Targeted disruption of the ATR kinase domain in mice.
- Analysis of ATR heterozygous (ATR+/-) mice and embryonic stem (ES) cells.
- Assessment of DNA-damaging agent sensitivity in ATR+/- ES cells.
Main Results:
- Targeted inactivation of ATR resulted in embryonic lethality before embryonic day 8.5.
- ATR heterozygous mice were fertile with no observable phenotype.
- ATR+/- ES cells showed no increased sensitivity to DNA-damaging agents, but loss of both alleles was lethal.
Conclusions:
- ATR is essential for early embryonic development in mammals.
- Unlike ATM, ATR has a critical, non-redundant function in early mammalian development.
- Complete loss of ATR function is incompatible with embryonic survival.
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