In vivo genome editing restores haemostasis in a mouse model of haemophilia

Li, Hojun, Haurigot, Virginia, Doyon, Yannick, Li, Tianjian, Wong, Sunnie Y., Bhagwat, Anand S., Malani, Nirav, Anguela, Xavier M., Sharma, Rajiv, Ivanciu, Lacramiora, Murphy, Samuel L., Finn, Jonathan D., Khazi, Fayaz R., Zhou, Shangzhen, Paschon, David E., Rebar, Edward J., Bushman, Frederic D., Gregory, Philip D., Holmes, Michael C., High, Katherine A.·2011·Nature

DOI 10.1038/nature10177

Cited by (549)

Items 201–300 of 549

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A D Diwan, Arun S. Ninawe, Sanjay N. Harke · 2017 · Canadian Journal of Biotechnology
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Delivery methods for site-specific nucleases: Achieving the full potential of therapeutic gene editing
Jia Liu, Sailan Shui · 2016 · Journal of Controlled Release
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Xian Li, Dangzhi Zhao, Xiaojun Hu · 2020 · Scientometrics
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Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.
Bryson Te, Caitlin M. Anglin, P. Hudson Bridges, Renee N. Cottle · 2017 · PubMed Central
Demographics, treatment patterns, safety, and real-world effectiveness in patients aged 70 years and over with chronic lymphocytic leukemia receiving bendamustine with or without rituximab: a retrospective study
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Pinki Dey, Arnab Bhattacherjee · 2018 · Soft Matter
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Proven and novel strategies for efficient editing of the human genome
Claudio Mussolino, Tafadzwa Mlambo, Toni Cathomen · 2015 · Current Opinion in Pharmacology
CRISPR technology for gene therapy
Katherine A. High, Philip D. Gregory, Charles A. Gersbach · 2014 · Nature Medicine
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