mRNA from TriLink Improves CRISPR/Cas9-mediated Gene Repair In Vivo
The development of therapeutic gene repair using CRISPR (clustered, regularly interspaced, short palindromic repeats) and Cas9 (CRISPR associated protein 9) has been problematic but is rapidly advancing. Published last month in Nature Biotechnology, Yin et al. report a potentially safe and effective method of CRISPR/Cas9-directed gene repair that utilizes a combination of TriLink mRNA and adeno-associated virus (AAV) to successfully correct gene alterations in vivo.
Though the potential of CRISPR/Cas9 system is vast, several challenges have persisted. These challenges include the development of an efficient delivery system for CRISPR/Cas9 components, increased efficiency of homology directed repair (HDR), and the reduction of off-target editing, all of which are critical for feasibility in the clinic.
To address these issues of efficiency and safety, the authors combined the viral and non-viral systems. They packaged TriLink Cas9 mRNA in lipid nanoparticles, single guide RNA (sgRNA) and...
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