CRISPR System with AAV
Zhang’s group from the Broad Institute (MIT) breed a SpCas9 knock-in mouse that can express SpCas9 constitutively or Cre-dependently in cells of whole body, and demonstrated that utilizing AAV delivering sgRNA and donor sequence can precisely modify the KRAS gene into a tumor-causing mutation, which then results in rapid tumor growth in 1 to 3 months (5). SpCas9 mouse is now available from Jackson Lab.
On the final day of 2015, three groups published their promising results on utilizing AAV to deliver Cas9 to therapy in DMD mouse as a therapeutics published in Science (8, 9, and 10). All three groups applied the same strategy: injection of AAV expressing SaCas9/SpCas9 and sgRNAs to delete the mutated Exon 23 in DMD mouse model, thereby restoring the truncated but still functional Dystrophin.
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