학술논문

Targeted nonviral delivery of genome editors in vivo
Document Type
article
Source
Proceedings of the National Academy of Sciences of the United States of America. 121(11)
Subject
Medical Biotechnology
Biomedical and Clinical Sciences
Biotechnology
Gene Therapy
Genetics
Development of treatments and therapeutic interventions
5.2 Cellular and gene therapies
Commerce
Gene Editing
Genetic Therapy
RNA
Messenger
Ribonucleoproteins
genome editing
CRISPR-Cas
in vivo delivery
nonviral delivery
targeted delivery
Language
Abstract
Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that will drive biological discovery and transform the field of cell and gene therapy. Here, we discuss recent advances in the delivery of CRISPR-Cas genome editors either as preassembled ribonucleoproteins or encoded in mRNA. Both strategies avoid pitfalls of viral vector-mediated delivery and offer advantages including transient editor lifetime and potentially streamlined manufacturing capability that are already proving valuable for clinical use. We review current applications and future opportunities of these emerging delivery approaches that could make genome editing more efficacious and accessible in the future.