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

Informazioni relative al progetto

BREATHE

ID dell’accordo di sovvenzione: 637752

DOI 10.3030/637752

Progetto chiuso

Data della firma CE 25 Marzo 2015

Data di avvio 1 Aprile 2015

Data di completamento 31 Marzo 2020

Finanziato da

• EXCELLENT SCIENCE - European Research Council (ERC)

Costo totale € 1 497 125,00

Contributo UE € 1 497 125,00

Coordinato da

EBERHARD KARLS UNIVERSITAET TUEBINGEN
Germany

Pubblicazioni

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

Autori: Michael S. D. Kormann, Alexander Dewerth, Felizitas Eichner, Praveen Baskaran, Andreas Hector, Nicolas Regamey, Dominik Hartl, Rupert Handgretinger, Justin S. Antony
Pubblicato in: PLOS ONE, Numero 12/8, 2017, Pagina/e e0183526, ISSN 1932-6203
Editore: Public Library of Science
DOI: 10.1371/journal.pone.0183526

The Oral and Craniofacial Relevance of Chemically Modified RNA Therapeutics

Autori: Satheesh Elangovan,corresponding author1 Michael S.D. Kormann,2 Behnoush Khorsand,3 and Aliasger K. Salemcorresponding author1,3
Pubblicato in: Discovery Medicine, 2016, ISSN 1539-6509
Editore: Discovery Medicine

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

Autori: 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
Pubblicato in: Scientific Reports, Numero 8/1, 2018, ISSN 2045-2322
Editore: Nature Publishing Group
DOI: 10.1038/s41598-018-34960-0

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

Autori: Itishri Sahu, A.K.M. Ashiqul Haque, Brian Weidensee, Petra Weinmann, Michael S.D. Kormann
Pubblicato in: Molecular Therapy, Numero 27/4, 2019, Pagina/e 803-823, ISSN 1525-0016
Editore: 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

Autori: 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
Pubblicato in: Molecular and Cellular Pediatrics, Numero 5/1, 2018, ISSN 2194-7791
Editore: Springer Open
DOI: 10.1186/s40348-018-0086-1

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

Autori: 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
Pubblicato in: Molecular Therapy - Nucleic Acids, Numero 12, 2018, Pagina/e 530-542, ISSN 2162-2531
Editore: Nature Publishing Group
DOI: 10.1016/j.omtn.2018.06.010

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

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

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

Autori: Justin S. Antony, Alexander Dewerth, Ashiqul Haque, Rupert Handgretinger, Michael S.D. Kormann
Pubblicato in: Molecular and Cellular Pediatrics, Numero 2/1, 2015, ISSN 2194-7791
Editore: SpringerOpen
DOI: 10.1186/s40348-015-0022-6

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

Autori: Franziska Zeyer, Benedikt Mothes, Clara Will, Melanie Carevic, Jennifer Rottenberger, Bernd Nürnberg, Dominik Hartl, Rupert Handgretinger, Sandra Beer-Hammer, Michael S. D. Kormann
Pubblicato in: PLOS ONE, Numero 11/4, 2016, Pagina/e e0154001, ISSN 1932-6203
Editore: Public Library of Science
DOI: 10.1371/journal.pone.0154001

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

Autori: 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
Pubblicato in: Nature Biotechnology, 2015, ISSN 1087-0156
Editore: Nature Publishing Group
DOI: 10.1038/nbt.3241