Targeted nonviral delivery of genome editors in vivo

• 成果类型: Article
• 署名作者: Tsuchida, Connor A.; Wasko, Kevin M.; Hamilton, Jennifer R.; Doudna, Jennifer A.
• 署名单位: University of California System; University of California Berkeley; University of California System; University of California Berkeley; University of California System; University of California Berkeley; University of California System; University of California Berkeley; University of California System; University of California Berkeley; United States Department of Energy (DOE); Lawrence Berkeley National Laboratory; University of California System; University of California San Francisco; The J David Gladstone Institutes; Howard Hughes Medical Institute; University of California System; University of California Berkeley
• 刊物名称: PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
• ISSN/ISSBN: 0027-8883
• DOI: 10.1073/pnas.2307796121
• 发表日期: 2024-03-12
• 关键词: cas9 ribonucleoprotein; extracellular vesicles; nanoparticle delivery; hematopoietic stem; highly efficient; messenger-rna; gene-therapy; cells; protein; crispr

摘要

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.