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Technology Overview

OHSU # 2391 — CRISPR/Cas9-targeted correction of heterozygous mutations in human preimplantation embryos

Technology Overview

More than 10,000 monogenic inherited disorders have been identified, affecting millions of people worldwide. Among these are the autosomal dominant mutations, where inheritance of a single copy of a defective gene can result in clinical symptoms. Dominant mutations that manifest as late-onset adult disorders include BRCA1 and BRCA2, associated with a high risk of breast and ovarian cancers and MYBPC3 causing hypertrophic cardiomyopathy (HCM).  OHSU has a proprietary method to correct a mutant allele of a gene of interest in an embryo allowing homology-directed repair to prevent transmission of monogenic inherited disorders such as HCM.

Licensing Opportunity

This technology is available for licensing and/or co-development.



Filed United States
Published United States WO 2018/195418
Published Mexico
Issued Singapore
Published United States US 2021/0130849 A1