Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
Broad Institute · Howard Hughes Medical Institute · +7 more institutions
Abstract
Methods to deliver gene editing agents in vivo as ribonucleoproteins could offer safety advantages over nucleic acid delivery approaches. We report the development and application of engineered DNA-free virus-like particles (eVLPs) that efficiently package and deliver base editor or Cas9 ribonucleoproteins. By engineering VLPs to overcome cargo packaging, release, and localization bottlenecks, we developed fourth-generation eVLPs that mediate efficient base editing in several primary mouse and human cell types. Using different glycoproteins in eVLPs alters their cellular tropism. Single injections of eVLPs into mice support therapeutic levels of base editing in multiple tissues, reducing serum Pcsk9 levels 78%…
Citation impact
- FWCI
- 100.29
- Percentile
- 100%
- References
- 96
Authors
14- SBSamagya Banskota
Broad Institute, Howard Hughes Medical Institute, Harvard University
- ARAditya Raguram
Broad Institute, Howard Hughes Medical Institute, Harvard University
- SSSusie Suh
University of California, Irvine, Case Western Reserve University
- SWSamuel W. Du
University of California, Irvine
- JRJessie R. Davis
Broad Institute, Howard Hughes Medical Institute, Harvard University
Topics & keywords
- Biology
- Genome editing
- Gene delivery
- In vivo
- Ribonucleoprotein
- CRISPR
- Guide RNA
- Cas9