In vivo prime editing of a metabolic liver disease in mice

Prime editing is a highly versatile CRISPR-based genome editing technology that works without DNA double-strand break formation. Despite rapid technological advances, in vivo application for the treatment of genetic diseases remains challenging. Here, we developed a size-reduced SpCas9 prime editor...

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Publicado en:Sci Transl Med
Main Authors: Böck, Desirée, Rothgangl, Tanja, Villiger, Lukas, Schmidheini, Lukas, Matsushita, Mai, Mathis, Nicolas, Ioannidi, Eleonora, Rimann, Nicole, Grisch-Chan, Hiu Man, Kreutzer, Susanne, Kontarakis, Zacharias, Kopf, Manfred, Thöny, Beat, Schwank, Gerald
Formato: Artigo
Idioma:Inglês
Publicado: 2022
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7614134/
https://ncbi.nlm.nih.gov/pubmed/35294257
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.abl9238
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