Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell Type Specific Gene Editing
Rouet et al., J Am Chem Soc. 2018 [PDF] [supp. info]
Chemical modification allows a Cas9 RNA-protein (RNP) complex to be selectively taken into cells bearing a liver-associated receptor. This demonstrates the feasibility of using molecular targeting to specify which cells Cas9 will edit, potentially for therapeutic use in vivo.
The Promise and Challenge of In Vivo Delivery for Genome Therapeutics
Ross Wilson & Luke Gilbert, ACS Chem Biol. 2018 [PDF]
A review of progress towards genome editing that can cure, prevent, or treat disease, along with a summary of the hurdles that remain.