Posts tagged 'modified RNA'

CleanCap® IVT Cas9 mRNA in Micelles Used for Genome Editing in Mouse Brain for the First Time

CRISPR-based genome editing tools show tremendous potential as therapeutic agents for the treatment of genetic diseases and viral infections, as discussed in previous Zone blogs and the June 2021 TriLink Research Spotlight titled, A Novel Base-Editing Strategy Promises to Treat Sickle Cell Disease. Nevertheless, safe and efficient delivery of CRISPR tools to target tissues continues to attract growing attention (see Chart). A wide variety of technical approaches are being explored, the main one being the formulation of nano-sized particles loaded with different types of CRISPR-related cargo.

1 year ago
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