New CRISPR “Cut-and-Paste” System Corrects Metabolic Liver Disease

Genetic and Engineering News 2 February, 2015

Scientists at the University of Pennsylvania have used an adeno-associated virus and CRISPR/Cas9 technology to correct a rare liver disease that results from a defect in the urea cycle.  Interestingly, the researchers found that the treatment was more effective in newborn over adult mice.  Further improvements of the efficiency of repaired DNA integration is needed before genetic treatments are available for humans.  The original research can be found at

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.

Leave a Reply