Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

Yin et al. Nat Biotechnol (2014) 32:551-553. http://www.ncbi.nlm.nih.gov/pubmed/24681508

Fatal hereditary diseases are potential targets for CRISPR/Cas9 gene therapies using homology directed repair.  Using a mouse model for hereditary tyrosinemia type I – which causes liver damage and ultimately failure due to the buildup of toxic metabolites in the tyrosine catabolic pathway – d containing the same G to A point mutation found in the human form of the disease, Yin et al where able to repair the gene and reverse the associated phenotypes.  While CRISPR/Cas9 gene editing has yet to be used to correct human genetic disease, results such as this provide a promising outlook for treatment of genetic diseases.

Author: Advanced Analytical

Advanced Analytical Technologies, Inc. (AATI) simplifies complex genomics workflows to accelerate research and discovery in pharmaceuticals, life science, biofuels, biotechnology and healthcare.

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