Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients

Schwank, G. Cell Stem Cell (2013) 13:653-658. http://www.ncbi.nlm.nih.gov/pubmed/24315439

CRISPR/Cas9 systems hold promise as a therapeutic gene therapy for a variety of chronic genetic diseases.  Cystic Fibrosis is the result of mutations in both alleles of the CTFR gene and is currently not a treatable condition.  In this study the authors present results demonstrating the ability of a CRISPR/Cas9 system to replace the mutant copy with a functional allele through homologous-end-joining repair in cultured small intestine cells of a cystic fibrosis patient.  The specificity of CRISPR/Cas9 systems could allow for gene therapies to cure 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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