Columbia Technology Ventures

Conditional knockout mouse model to study genomic instability of BRCA1 mutations

This technology is a mouse model for conditional knockout of part of the BRCA1 gene, for use in studying molecular mechanisms of development of human hereditary breast and ovarian cancers.

Unmet Need: Animal model to study mechanisms of tumorigenesis in BRCA1 mutations

Although 7% of human hereditary breast and ovarian cancers are caused by mutations in BRCA1, the exact mechanism of how BRCA1 mutation leads to tumorigenesis remains unclear. Current methods have sought to define the regions of conserved domains essential for maintaining BRCA1’s role in maintaining genomic stability. Studies have shown that mutations in the DNA damage response factor 53BP1 rescues genomic stability in BRCA1 mutants, but the mechanism of this regulation remains unclear and there are no clear genetic models to assess this genetic interplay.

The Technology: Conditional BRCA1 mutated mouse strain for studying human cancer tumorigenesis

This technology describes a mouse line with inducible knockout of exon 2 of BRCA1. Upon loss of exon 2, this gene encodes a mutant protein analogous to the “RING-less” BRCA1 protein implicated in human hereditary cancer. The BRCA1 mutation here may be combined with mutations in other DNA damage response factors to allow researchers to further study how genomic instability and ultimately tumorigenesis emerges. The conditional nature of this allele allows combination with other mouse lines to test therapeutics and mechanisms of tumorigenesis. This technology provides a conditional approach to targeting BRCA1 mutation in mice, with implications to further understand and treat ovarian and breast cancers.

This technology has been validated to conditionally knockdown BRCA1 in a mouse model and with western blot and immunofluorescence of mouse embryonic fibroblasts.

Applications:

  • Diagnostic assays for breast and ovarian cancer
  • Drug screening tool for breast and ovarian cancer
  • Research tool for analysis of mechanisms of genomic instability, involving BRCA1

Advantages:

  • Conditional expression of embryonic lethal gene mutation
  • Uses standard genetic technologies for creating the mouse model
  • Recapitulates common human cancer mutation
  • Compatible with crossing to other conditional transgenic mutants

Lead Inventor:

Richard Baer, Ph.D.

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