CRISPR corrects X-linked granulomatous disease in a mouse model

Andrew Han, GenomeWeb, https://www.genomeweb.com/gene-silencinggene-editing/crispr-corrects-genetic-immunodeficiency-blood-stem-cells

In a new article published in the journal Science Translational Medicine (http://stm.sciencemag.org/content/9/372/eaah3480), researchers used CRISPR to correct a mutation in NOX2 that causes X-linked granulomatous disease ex vivo, followed by implantation into a mouse model.  Mice implanted with the edited cells demonstrated functional NOX2 protein for up to 5 months, demonstrating the potential CRISPR has in treating genetic disorders.

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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