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Small molecule screen employing patient-derived iPS hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin Deficiency
Deniz Kent, Soon Seng Ng, Payam Khoshkenar, Adam M. Syanda, Chao Zheng Li, Marina Zieger, Cindy Greer, Stephanie Hatch, Joe Segal, Samuel J.I. Blackford, Vivek Chowdary, Taylor Ismali, Davide Danovi, Sunil Sahdeo, View ORCID ProfileDaniel Ebner, Christian Mueller, S. Tamir Rashid
doi: https://doi.org/10.1101/2021.09.17.460732
Deniz Kent
2Centre for Stem Cells and Regenerative Medicine, King’s College London; London SE1 9RT, United Kingdom
3Gene Therapy Center, University of Massachusetts; Worchester, Massachusetts 01655, United States
Soon Seng Ng
1Metabolism and Hepatology and Gastroenterology, Imperial College London; London W12 0NN, United Kingdom
Payam Khoshkenar
3Gene Therapy Center, University of Massachusetts; Worchester, Massachusetts 01655, United States
Adam M. Syanda
1Metabolism and Hepatology and Gastroenterology, Imperial College London; London W12 0NN, United Kingdom
Chao Zheng Li
2Centre for Stem Cells and Regenerative Medicine, King’s College London; London SE1 9RT, United Kingdom
Marina Zieger
3Gene Therapy Center, University of Massachusetts; Worchester, Massachusetts 01655, United States
Cindy Greer
3Gene Therapy Center, University of Massachusetts; Worchester, Massachusetts 01655, United States
Stephanie Hatch
5National Phenotypic Screening Centre, University of Oxford; Headington, Oxford OX3 7FZ, United Kingdom
Joe Segal
2Centre for Stem Cells and Regenerative Medicine, King’s College London; London SE1 9RT, United Kingdom
Samuel J.I. Blackford
1Metabolism and Hepatology and Gastroenterology, Imperial College London; London W12 0NN, United Kingdom
Vivek Chowdary
3Gene Therapy Center, University of Massachusetts; Worchester, Massachusetts 01655, United States
Taylor Ismali
4Discovery Sciences, Janssen Research and Development; San Diego, California 92121, United States
Davide Danovi
2Centre for Stem Cells and Regenerative Medicine, King’s College London; London SE1 9RT, United Kingdom
Sunil Sahdeo
4Discovery Sciences, Janssen Research and Development; San Diego, California 92121, United States
Daniel Ebner
5National Phenotypic Screening Centre, University of Oxford; Headington, Oxford OX3 7FZ, United Kingdom
Christian Mueller
3Gene Therapy Center, University of Massachusetts; Worchester, Massachusetts 01655, United States
S. Tamir Rashid
1Metabolism and Hepatology and Gastroenterology, Imperial College London; London W12 0NN, United Kingdom
Article usage
Posted September 17, 2021.
Small molecule screen employing patient-derived iPS hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin Deficiency
Deniz Kent, Soon Seng Ng, Payam Khoshkenar, Adam M. Syanda, Chao Zheng Li, Marina Zieger, Cindy Greer, Stephanie Hatch, Joe Segal, Samuel J.I. Blackford, Vivek Chowdary, Taylor Ismali, Davide Danovi, Sunil Sahdeo, Daniel Ebner, Christian Mueller, S. Tamir Rashid
bioRxiv 2021.09.17.460732; doi: https://doi.org/10.1101/2021.09.17.460732
Small molecule screen employing patient-derived iPS hepatocytes identifies LRRK2 as a novel therapeutic target for Alpha1 Antitrypsin Deficiency
Deniz Kent, Soon Seng Ng, Payam Khoshkenar, Adam M. Syanda, Chao Zheng Li, Marina Zieger, Cindy Greer, Stephanie Hatch, Joe Segal, Samuel J.I. Blackford, Vivek Chowdary, Taylor Ismali, Davide Danovi, Sunil Sahdeo, Daniel Ebner, Christian Mueller, S. Tamir Rashid
bioRxiv 2021.09.17.460732; doi: https://doi.org/10.1101/2021.09.17.460732
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