Gene Editing without Cutting DNA
Salk Institute scientists have demonstrated how it’s possible to use two genetically engineered AAV vectors that result is altering a gene’s activity without cutting its double stranded DNA. “The researchers inserted the gene for the Cas9 enzyme into one AAV virus. They used another AAV virus to introduce a short single guide RNA (sgRNA), which specifies the precise location in the mouse genome where Cas9 will bind, and a transcriptional activator. The shorter sgRNA is only 14 or 15 nucleotides compared with the standard 20 nucleotides used in most CRISPR-Cas9 techniques, and this prevents Cas9 from cutting the DNA.” This approach was successful in reversing kidney damage in mouse models of that disease. No mention of immune response to sgRNA. MORE
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