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.