Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

@article{Ling2021LentiviralDO,
  title={Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice},
  author={Sikai Ling and Shiqi Yang and Xinde Hu and Di Yin and Yao Dai and Xiaoqin Qian and Dawei Wang and Xiaoyong Pan and Jiaxu Hong and Xiaodong Sun and Hui Yang and Soren Riis Paludan and Yujia Cai},
  journal={Nature Biomedical Engineering},
  year={2021},
  pages={1-13}
}
Therapeutic genome editing requires effective and targeted delivery methods. The delivery of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy, but can cause sustained Cas9 expression, anti-Cas9 immune responses and off-target edits. Lentiviral vectors have been engineered to deliver nucleases that are expressed transiently, but in vivo evidence of their biomedical efficacy is lacking. Here, we show that the lentiviral codelivery of Streptococcus pyogenes… Expand
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