PBAE-Cas9 nanoparticles

PBAE-Cas9 nanoparticles are a non-viral, research-stage gene-editing delivery system that utilizes poly(β-amino ester) (PBAE) polymers to encapsulate and deliver CRISPR/Cas9 components into target cells. The PBAE polymer serves as a biodegradable, cationic carrier designed to facilitate endosomal escape and the subsequent cytosolic delivery of the Cas9 gene-editing machinery, which can be formulated as plasmid DNA, mRNA, or ribonucleoproteins (RNPs). This platform is being investigated for a variety of therapeutic applications, including the treatment of sickle cell disease (SCD) by targeting the BCL11A enhancer in hematopoietic stem cells (HSCs), and the treatment of cervical cancer by targeting HPV oncogenes such as HPV16 E7. The system is noted for providing a potentially less toxic alternative to electroporation for ex vivo gene editing of primary human cells.

Other names

poly(beta-amino ester) Cas9 nanoparticlesPBAE-Cas9 NPPBAE-Cas-9 NPPBAE-Cas 9 NPpolymeric Cas9 nanoparticles