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

Technologies that can safely edit genes in the brains of adult animals may revolutionize the treatment of neurological diseases and the understanding of brain function. Here, we demonstrate that intracranial injection of CRISPR–Gold, a nonviral delivery vehicle for the CRISPR–Cas9 ribonucleoprotein,...

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Dades bibliogràfiques
Publicat a:Nat Biomed Eng
Autors principals: Lee, Bumwhee, Lee, Kunwoo, Panda, Shree, Gonzales-Rojas, Rodrigo, Chong, Anthony, Bugay, Vladislav, Park, Hyo Min, Brenner, Robert, Murthy, Niren, Lee, Hye Young
Format: Artigo
Idioma:Inglês
Publicat: 2018
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6544395/
https://ncbi.nlm.nih.gov/pubmed/30948824
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41551-018-0252-8
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