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Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
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Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

Villiger, Lukas ; Grisch-Chan, Hiu Man ; Lindsay, Helen ; Ringnalda, Femke ; Pogliano, Chiara B ; Allegri, Gabriella ; Fingerhut, Ralph ; Häberle, Johannes ; Matos, Joao ; Robinson, Mark D ; Thöny, Beat ; Schwank, Gerald

Nature medicine, 2018-10, Vol.24 (10), p.1519-1525 [Periódico revisado por pares]

United States: Nature Publishing Group

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Core Outcome Set-STAndardised Protocol Items: the COS-STAP Statement
Core Outcome Set-STAndardised Protocol Items: the COS-STAP Statement
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Core Outcome Set-STAndardised Protocol Items: the COS-STAP Statement

Kirkham, Jamie J ; Gorst, Sarah ; Altman, Douglas G ; Blazeby, Jane M ; Clarke, Mike ; Tunis, Sean ; Williamson, Paula R

Current controlled trials in cardiovascular medicine, 2019-02, Vol.20 (1), p.116-116, Article 116 [Periódico revisado por pares]

England: BioMed Central Ltd

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3
In utero CRISPR-mediated therapeutic editing of metabolic genes
In utero CRISPR-mediated therapeutic editing of metabolic genes
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In utero CRISPR-mediated therapeutic editing of metabolic genes

Rossidis, Avery C ; Stratigis, John D ; Chadwick, Alexandra C ; Hartman, Heather A ; Ahn, Nicholas J ; Li, Haiying ; Singh, Kshitiz ; Coons, Barbara E ; Li, Li ; Lv, Wenjian ; Zoltick, Philip W ; Alapati, Deepthi ; Zacharias, William ; Jain, Rajan ; Morrisey, Edward E ; Musunuru, Kiran ; Peranteau, William H

Nature medicine, 2018-10, Vol.24 (10), p.1513-1518 [Periódico revisado por pares]

United States: Nature Publishing Group

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4
COMPare: a prospective cohort study correcting and monitoring 58 misreported trials in real time
COMPare: a prospective cohort study correcting and monitoring 58 misreported trials in real time
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COMPare: a prospective cohort study correcting and monitoring 58 misreported trials in real time

Goldacre, Ben ; Drysdale, Henry ; Dale, Aaron ; Milosevic, Ioan ; Slade, Eirion ; Hartley, Philip ; Marston, Cicely ; Powell-Smith, Anna ; Heneghan, Carl ; Mahtani, Kamal R

Current controlled trials in cardiovascular medicine, 2019-02, Vol.20 (1), p.118-118, Article 118 [Periódico revisado por pares]

England: BioMed Central Ltd

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5
RNA editing in the forefront of epitranscriptomics and human health
RNA editing in the forefront of epitranscriptomics and human health
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RNA editing in the forefront of epitranscriptomics and human health

Christofi, Theodoulakis ; Zaravinos, Apostolos

Journal of translational medicine, 2019-09, Vol.17 (1), p.319-319, Article 319 [Periódico revisado por pares]

England: BioMed Central Ltd

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6
Mini-dCas13X-mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy
Mini-dCas13X-mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy
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Mini-dCas13X-mediated RNA editing restores dystrophin expression in a humanized mouse model of Duchenne muscular dystrophy

Li, Guoling ; Jin, Ming ; Li, Zhifang ; Xiao, Qingquan ; Lin, Jiajia ; Yang, Dong ; Liu, Yuanhua ; Wang, Xing ; Xie, Long ; Ying, Wenqin ; Wang, Haoqiang ; Zuo, Erwei ; Shi, Linyu ; Wang, Ning ; Chen, Wanjin ; Xu, Chunlong ; Yang, Hui

The Journal of clinical investigation, 2023-02, Vol.133 (3), p.1-14 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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7
Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy
Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy
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Nonviral base editing of KCNJ13 mutation preserves vision in a model of inherited retinal channelopathy

Kabra, Meha ; Shahi, Pawan K ; Wang, Yuyuan ; Sinha, Divya ; Spillane, Allison ; Newby, Gregory A ; Saxena, Shivani ; Tong, Yao ; Chang, Yu ; Abdeen, Amr A ; Edwards, Kimberly L ; Theisen, Cole O ; Liu, David R ; Gamm, David M ; Gong, Shaoqin ; Saha, Krishanu ; Pattnaik, Bikash R

The Journal of clinical investigation, 2023-10, Vol.133 (19), p.1-15 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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8
The JCI Scholar experience strengthens physician-scientist training
The JCI Scholar experience strengthens physician-scientist training
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The JCI Scholar experience strengthens physician-scientist training

Vasavda, Chirag ; Uddin, Olivia ; Lee, Michael S

The Journal of clinical investigation, 2021-03, Vol.131 (6), p.1-2 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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9
Introducing the new CONSORT extension for stepped-wedge cluster randomised trials
Introducing the new CONSORT extension for stepped-wedge cluster randomised trials
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Introducing the new CONSORT extension for stepped-wedge cluster randomised trials

Hemming, Karla ; Taljaard, Monica ; Grimshaw, Jeremy

Current controlled trials in cardiovascular medicine, 2019-01, Vol.20 (1), p.68-68, Article 68 [Periódico revisado por pares]

England: BioMed Central Ltd

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10
Make it even better
Make it even better
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Make it even better

McNally, Elizabeth M

The Journal of clinical investigation, 2022-03, Vol.132 (5), p.1-2 [Periódico revisado por pares]

United States: American Society for Clinical Investigation

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