Biochemically modified messenger RNA encoding nucleases for in vivo gene correction of severe inherited lung diseases

Project Information

BREATHE

Grant agreement ID: 637752

DOI

10.3030/637752

Project closed

EC signature date 25 March 2015

Start date 1 April 2015

End date 31 March 2020

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost

€ 1 497 125,00

EU contribution

€ 1 497 125,00

Coordinated by

EBERHARD KARLS UNIVERSITAET TUEBINGEN
Germany

CORDIS provides links to public deliverables and publications of HORIZON projects.

Links to deliverables and publications from FP7 projects, as well as links to some specific result types such as dataset and software, are dynamically retrieved from OpenAIRE .

Publications

Peer reviewed articles (10)

Transcriptomic profile of cystic fibrosis patients identifies type I interferon response and ribosomal stalk proteins as potential modifiers of disease severity

Author(s): Michael S. D. Kormann, Alexander Dewerth, Felizitas Eichner, Praveen Baskaran, Andreas Hector, Nicolas Regamey, Dominik Hartl, Rupert Handgretinger, Justin S. Antony
Published in: PLOS ONE, Issue 12/8, 2017, Page(s) e0183526, ISSN 1932-6203
Publisher: Public Library of Science
DOI: 10.1371/journal.pone.0183526

The Oral and Craniofacial Relevance of Chemically Modified RNA Therapeutics

Author(s): Satheesh Elangovan,corresponding author1 Michael S.D. Kormann,2 Behnoush Khorsand,3 and Aliasger K. Salemcorresponding author1,3
Published in: Discovery Medicine, 2016, ISSN 1539-6509
Publisher: Discovery Medicine

Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis

Author(s): A. K. M. Ashiqul Haque, Alexander Dewerth, Justin S. Antony, Joachim Riethmüller, Georg R. Schweizer, Petra Weinmann, Ngadhnjim Latifi, Hanzey Yasar, Nicoletta Pedemonte, Elvira Sondo, Brian Weidensee, Anjali Ralhan, Julie Laval, Patrick Schlegel, Christian Seitz, Brigitta Loretz, Claus-Michael Lehr, Rupert Handgretinger, Michael S. D. Kormann
Published in: Scientific Reports, Issue 8/1, 2018, ISSN 2045-2322
Publisher: Nature Publishing Group
DOI: 10.1038/s41598-018-34960-0

Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases

Author(s): Itishri Sahu, A.K.M. Ashiqul Haque, Brian Weidensee, Petra Weinmann, Michael S.D. Kormann
Published in: Molecular Therapy, Issue 27/4, 2019, Page(s) 803-823, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2019.02.019

Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors

Author(s): Justin S. Antony, Ngadhnjim Latifi, A. K. M. Ashiqul Haque, Andrés Lamsfus-Calle, Alberto Daniel-Moreno, Sebastian Graeter, Praveen Baskaran, Petra Weinmann, Markus Mezger, Rupert Handgretinger, Michael S. D. Kormann
Published in: Molecular and Cellular Pediatrics, Issue 5/1, 2018, ISSN 2194-7791
Publisher: Springer Open
DOI: 10.1186/s40348-018-0086-1

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

Author(s): Sriram Vaidyanathan, Krist T. Azizian, A.K.M. Ashiqul Haque, Jordana M. Henderson, Ayal Hendel, Sabrina Shore, Justin S. Antony, Richard I. Hogrefe, Michael S.D. Kormann, Matthew H. Porteus, Anton P. McCaffrey
Published in: Molecular Therapy - Nucleic Acids, Issue 12, 2018, Page(s) 530-542, ISSN 2162-2531
Publisher: Nature Publishing Group
DOI: 10.1016/j.omtn.2018.06.010

RNA ImmunoGenic Assay: Simple method for detecting immunogenicity of in vitro transcribed mRNA

Author(s): AKM Ashiqul Haque, Petra Weinmann, Sumit Biswas, Rupert Handgretinger, Markus Mezger, Michael S. D. Kormann, Justin S. Antony
Published in: ADVANCES IN CELL AND GENE THERAPY, Issue 3/2, 2020, ISSN 2573-8461
Publisher: John Wiley & Sons Ltd.
DOI: 10.1002/acg2.79

Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies

Author(s): Justin S. Antony, Alexander Dewerth, Ashiqul Haque, Rupert Handgretinger, Michael S.D. Kormann
Published in: Molecular and Cellular Pediatrics, Issue 2/1, 2015, ISSN 2194-7791
Publisher: SpringerOpen
DOI: 10.1186/s40348-015-0022-6

mRNA-Mediated Gene Supplementation of Toll-Like Receptors as Treatment Strategy for Asthma In Vivo

Author(s): Franziska Zeyer, Benedikt Mothes, Clara Will, Melanie Carevic, Jennifer Rottenberger, Bernd Nürnberg, Dominik Hartl, Rupert Handgretinger, Sandra Beer-Hammer, Michael S. D. Kormann
Published in: PLOS ONE, Issue 11/4, 2016, Page(s) e0154001, ISSN 1932-6203
Publisher: Public Library of Science
DOI: 10.1371/journal.pone.0154001

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

Author(s): Azita J Mahiny, Alexander Dewerth, Lauren E Mays, Mohammed Alkhaled, Benedikt Mothes, Emad Malaeksefat, Brigitta Loretz, Jennifer Rottenberger, Darina M Brosch, Philipp Reautschnig, Pacharapan Surapolchai, Franziska Zeyer, Andrea Schams, Melanie Carevic, Martina Bakele, Matthias Griese, Matthias Schwab, Bernd Nürnberg, Sandra Beer-Hammer, Rupert Handgretinger, Dominik Hartl, Claus-Michael Lehr, M
Published in: Nature Biotechnology, 2015, ISSN 1087-0156
Publisher: Nature Publishing Group
DOI: 10.1038/nbt.3241