Paper of the field n°7
Click here to read more about the new method that speeds up gene editing by using packaged CRISPR–Cas9 tools.!
Abstract for General public
Genome editing with the use of the CRISPR-Cas9 system is a method that has the potential to cure genetic diseases. Effective genome editing requires a sufficient dose of Cas9 to enter cells. However, high doses may lead to undesirable side effects, such as off-target editing, cytotoxicity, and immune responses. Currently, the clinical use of Cas9 involves electroporation to introduce it into the cells. Scientists compared two delivery strategies—electroporation and packaged delivery via enveloped delivery vehicles (EDVs)—to investigate the dosage requirements for efficient genome editing. This study found that EDV-mediated editing was ~ 30 times more efficient than electroporation. Furthermore, EDV-based editing occurred at least twice as fast at the same dosage compared to electroporation. The researchers hypothesize that the accelerated genome editing observed with EDV delivery is due to the prolonged presence of Cas9 in the nucleus. This study suggests that packaged delivery could significantly reduce the required dose of Cas9 in clinical applications.
Abstract written by Anna Misiukiewicz from Institute of Bioorganic Chemistry of the Polish Academy of Sciences (Poland)
Karp et al. « Packaged delivery of CRISPR–Cas9 ribonucleoproteins accelerates genome editing. ». Nucleic Acids Research 53.5 (2025): gkaf105. https://academic.oup.com/nar/article/53/5/gkaf105/8044958
