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Nature Biomedical Engineering (1)

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Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours

Gene editing of a single gene in the brain of an adult mouse model of fragile X syndrome, achieved via the intracranial injection of a nonviral Cas9 delivery vehicle, rescues mice from the exaggerated repetitive behaviours caused by the disease.

Bumwhee Lee
Kunwoo Lee
Hye Young Lee
Research25 Jun 2018
Nature Biomedical Engineering

Volume: 2, P: 497-507

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