Technical Report abstract


Nature Medicine 14, 875 - 881 (2008)
Published online: 6 July 2008 | Corrected online: 11 July 2008 | doi:10.1038/nm.1719

Mouse embryonic stem cell–based functional assay to evaluate mutations in BRCA2

Sergey G Kuznetsov1, Pentao Liu1,2 & Shyam K Sharan1


Individuals with mutations in breast cancer susceptibility genes BRCA1 and BRCA2 have up to an 80% risk of developing breast cancer by the age of 70. Sequencing-based genetic tests are now available to identify mutation carriers in an effort to reduce mortality through prevention and early diagnosis. However, lack of a suitable functional assay hinders the risk assessment of more than 1,900 BRCA1 and BRCA2 variants in the Breast Cancer Information Core database that do not clearly disrupt the gene product. We have established a simple, versatile and reliable assay to test for the functional significance of mutations in BRCA2 using mouse embryonic stem cells (ES cells) and bacterial artificial chromosomes and have used it to classify 17 sequence variants. The assay is based on the ability of human BRCA2 to complement the loss of endogenous Brca2 in mouse ES cells. This technique may also serve as a paradigm for functional analysis of mutations found in other genes linked to human diseases.

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  1. Mouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute at Frederick, 1050 Boyles Street, Frederick, Maryland 21702, USA.
  2. Current address: Wellcome Trust Sanger Institute, Wellcome Trust Genome Campus, Hinxton, Cambridge CB10 1HH, UK.

Correspondence to: Shyam K Sharan1 e-mail: sharans@mail.nih.gov

* In the version of this article initially published online, the online publication date was listed in correctly as 6 July 2007 on the PDF. The error has been corrected for all versions of this article.

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