Clinical Applications of CRISPR-based Genome Editing and Diagnostics
Dana Foss, Megan Hochstrasser, Ross Wilson; Transfusion 2019 [PDF]
We review CRISPR’s transition into the clinic, via ex vivo therapies and emerging diagnostic tools.
Engineering CRISPR-Cas9 RNA–Protein Complexes for Improved Function and Delivery
Romain Rouet, Lorena de Oñate, Jie Li, Niren Murthy, Ross Wilson, CRISPR J. 2018 [PDF]
We summarize recent progress in the engineering of Cas9 RNA-protein (RNP) complexes for cellular delivery, improved function, and more.
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.
Emerging Strategies for Genome Editing in the Brain
Dana Foss & Ross Wilson; Trends Mol Med. 2018 [PDF]
This Spotlight article describes nanoparticle-driven delivery of Cas9 RNP enzymes and compares/contrasts this approach to virally mediated delivery.