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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 exp...

Introduction   Synthetic mRNA produced by in vitro transcription (IVT) is the active pharmaceutical ingredient of approved vaccines and of many drugs under development. IVT mRNA typically contains several hundred bases of non-coding untranslated regions (UTRs) that are involved in the stabilization and translation of the mRNA. Remarkably, new research has discovered that these UTRs can be virtually eliminated without compromising translation of the encoded protein of interest. ...

Historically,  reporter genes  have been delivered as DNA plasmids for transcription into mRNA before translation into a reporter protein. In biological systems, ideal reporter proteins generate highly sensitive signals that are easily measurable. Thus, they are often  photoluminescent  (e.g., GFP) or  chemiluminescent  (e.g., luciferase).