Therapeutics

The Promise of Base Editing: Potential Applications in Science and Medicine

The discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system has revolutionized the field of molecular biology and medicine (reviewed by Kantor et al.). CRISPR-mediated genome editing initially involved the generation of a Cas9-induced double-strand DNA repaired by either non-homologous end joining (NHEJ) mechanisms, or by homology-directed repair (HDR).

The discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system has revolutionized the field of molecular biology and medicine (reviewed by Kantor et al.). CRISPR-mediated genome editing initially involved the generation of a Cas9-induced double-strand DNA (dsDNA) break (Figure 1) repaired by either non-homologous end joining (NHEJ) mechanisms, or by homology-directed repair (HDR).

6 months ago
252 view(s)

Applying CleanCap® mRNA: 4 Example Studies

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).

8 months ago
244 view(s)

Four Pillars for Successful Self-Amplifying RNA Vaccines…

The current universe of vaccines can be divided into two main categories, conventional vaccines and nucleic acid vaccines (Blakney et al.). Conventional vaccines are based on a protein adjuvant, and nucleic acid vaccines can be based on DNA or RNA. For this blog, we will focus on nucleic acid vaccines based on RNA structures and, in particular, self-amplifying RNA vaccines (Figure 1).

9 months ago
428 view(s)

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.

10 months ago
109 view(s)

Plasmids—Part 2: Perfecting Plasmid Production for IVT mRNA Manufacturing

This post is Part 2 of a series of Zone blogs featuring DNA plasmids used to produce in vitro transcribed (IVT) mRNA. Part 1, posted on June 8, 2021, provided historical perspectives on the discovery of plasmids, as well as descriptions of how these circular double-stranded DNAs enabled recombinant DNA technology and now IVT mRNA production. The two growth phases of plasmid applications are reflected in this chart of publications in PubMed indexed to gene therapy and genetic vaccination for two 10-year periods: 1991–2000 and 2012–2021, respectively.

11 months ago
447 view(s)
""