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Posts tagged 'gene editing'

Targeted Delivery of mRNA Therapeutics—4 Recent Examples
Zone in with Zon for 2022 Blog Highlights
Combating HIV/AIDS with mRNA Vaccines and CRISPR Gene Editing
Rare Disease Awareness Month and Day, February 28th
Spotlight on Women and Girls in Science
The Promise of Base Editing: Potential Applications in Science and Medicine
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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).

Applying CleanCap® mRNA: 4 Example Studies
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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).

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