CRISPR/Cas9-targeted correction of heterozygous mutations in human preimplantation embryos
OHSU # 2391
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.
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Senior Technology Development Manager