Search results for: 'mrna sea'

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. ...

This blog provides some perspective on published strategies for this type of sequence optimization, which is alternatively referred to sequence engineering. This topic has been trending upwards in recent years, as indicated by the linear forecast dashed line shown in this chart reports found by word searching the NIH PubMed database for 2013 – 2020.