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

Publikacje

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

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

The Oral and Craniofacial Relevance of Chemically Modified RNA Therapeutics

Autorzy: Satheesh Elangovan,corresponding author1 Michael S.D. Kormann,2 Behnoush Khorsand,3 and Aliasger K. Salemcorresponding author1,3
Opublikowane w: Discovery Medicine, 2016, ISSN 1539-6509
Wydawca: Discovery Medicine

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

Autorzy: 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
Opublikowane w: Scientific Reports, Issue 8/1, 2018, ISSN 2045-2322
Wydawca: Nature Publishing Group
DOI: 10.1038/s41598-018-34960-0

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

Autorzy: Itishri Sahu, A.K.M. Ashiqul Haque, Brian Weidensee, Petra Weinmann, Michael S.D. Kormann
Opublikowane w: Molecular Therapy, Issue 27/4, 2019, Page(s) 803-823, ISSN 1525-0016
Wydawca: 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

Autorzy: 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
Opublikowane w: Molecular and Cellular Pediatrics, Issue 5/1, 2018, ISSN 2194-7791
Wydawca: Springer Open
DOI: 10.1186/s40348-018-0086-1

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

Autorzy: 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
Opublikowane w: Molecular Therapy - Nucleic Acids, Issue 12, 2018, Page(s) 530-542, ISSN 2162-2531
Wydawca: Nature Publishing Group
DOI: 10.1016/j.omtn.2018.06.010

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

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

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

Autorzy: Justin S. Antony, Alexander Dewerth, Ashiqul Haque, Rupert Handgretinger, Michael S.D. Kormann
Opublikowane w: Molecular and Cellular Pediatrics, Issue 2/1, 2015, ISSN 2194-7791
Wydawca: SpringerOpen
DOI: 10.1186/s40348-015-0022-6

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

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

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

Autorzy: 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
Opublikowane w: Nature Biotechnology, 2015, ISSN 1087-0156
Wydawca: Nature Publishing Group
DOI: 10.1038/nbt.3241

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