Monday, September 22, 2014

Paper demonstrating #CRIPSR to correct human beta-thalassemia mutations in vitro.

  This made use of the piggyBAC transposon system to select for inserted clones; of the Puro+ clones, ~23% had undergone homologous recombination at HBB; of these, about 75% had recombined in such a way as to replace a mutation with wild type sequence.   

The piggyBAC transposon can be excised cleanly after the fact, allowing in vitro selection with a "clean getaway".   So this is very nice for certain in vitro applications. 
Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac. Xie FYe LChang JCBeyer AIWang JMuench MO, Kan YW.  Genome Res. 2014 Sep;24(9):1526-33. doi: 10.1101/gr.173427.114. Epub 2014 Aug 5.

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