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‘Hit and run’ genome editing forestalls macular degeneration

Lentiviruses co-packaging SpCas9 mRNA and an expression cassette encoding for a guide RNA targeting the Vegfa gene prevent mice from developing wet age-related macular degeneration induced by Vegfa.
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Pep Pàmies
Chief Editor, Nature Biomedical Engineering, Nature Research
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Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice - Nature Biomedical Engineering

Lentiviruses co-packaging SpCas9 mRNA and an expression cassette encoding for a guide RNA targeting the Vegfa gene prevent mice from developing wet age-related macular degeneration induced by Vegfa. [Summary amended to correct the description of the packaged cargo.]
Go to the profile of Pep Pàmies
Pep Pàmies
Chief Editor, Nature Biomedical Engineering, Nature Research

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Go to the profile of Pep Pàmies
Pep Pàmies
about 2 years ago

The cover illustrates that lentiviruses co-packaging SpCas9 mRNA and an expression cassette encoding for a guide RNA can transiently disrupt targeted disease-modifying genes.

See Ling et al.

Image: Yujia Cai and Sikai Ling, Shanghai Jiao Tong University. Cover design: Alex Wing.

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