A Small-Molecule-Responsive Riboswitch Enables Conditional Induction of Viral Vector-Mediated Gene Expression in Mice-Reference-Cited by-同舟云学术

A Small-Molecule-Responsive Riboswitch Enables Conditional Induction of Viral Vector-Mediated Gene Expression in Mice

Published:2020-05-19 Issue:6 Volume:9 Page:1292-1305
ISSN:2161-5063
Container-title:ACS Synthetic Biology
language:en
Short-container-title:ACS Synth. Biol.

Author:

Strobel Benjamin¹^ORCID,Düchs Matthias J.¹,Blazevic Dragica¹,Rechtsteiner Philipp¹,Braun Clemens²,Baum-Kroker Katja S.²,Schmid Bernhard²,Ciossek Thomas¹,Gottschling Dirk³,Hartig Jörg S.⁴^ORCID,Kreuz Sebastian¹^ORCID

Affiliation:

1. Research Beyond Borders, Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach an der Riss, 88397, Germany

2. Drug Discovery Sciences, Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach an der Riss, 88397, Germany

3. Medicinal Chemistry, Boehringer Ingelheim Pharma GmbH & Co. KG, Biberach an der Riss, 88397, Germany

4. Department of Chemistry, University of Konstanz, Konstanz, 78464, Germany

Publisher

American Chemical Society (ACS)

Subject

Biochemistry, Genetics and Molecular Biology (miscellaneous),Biomedical Engineering,General Medicine

Link

http://pubs.acs.org/doi/pdf/10.1021/acssynbio.9b00410

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1. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

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5. Next-generation muscle-directed gene therapy by in silico vector design

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