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

Publicaciones

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

Autores: Michael S. D. Kormann, Alexander Dewerth, Felizitas Eichner, Praveen Baskaran, Andreas Hector, Nicolas Regamey, Dominik Hartl, Rupert Handgretinger, Justin S. Antony
Publicado en: PLOS ONE, Edición 12/8, 2017, Página(s) e0183526, ISSN 1932-6203
Editor: Public Library of Science
DOI: 10.1371/journal.pone.0183526

The Oral and Craniofacial Relevance of Chemically Modified RNA Therapeutics

Autores: Satheesh Elangovan,corresponding author1 Michael S.D. Kormann,2 Behnoush Khorsand,3 and Aliasger K. Salemcorresponding author1,3
Publicado en: Discovery Medicine, 2016, ISSN 1539-6509
Editor: Discovery Medicine

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

Autores: 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
Publicado en: Scientific Reports, Edición 8/1, 2018, ISSN 2045-2322
Editor: Nature Publishing Group
DOI: 10.1038/s41598-018-34960-0

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

Autores: Itishri Sahu, A.K.M. Ashiqul Haque, Brian Weidensee, Petra Weinmann, Michael S.D. Kormann
Publicado en: Molecular Therapy, Edición 27/4, 2019, Página(s) 803-823, ISSN 1525-0016
Editor: 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

Autores: 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
Publicado en: Molecular and Cellular Pediatrics, Edición 5/1, 2018, ISSN 2194-7791
Editor: Springer Open
DOI: 10.1186/s40348-018-0086-1

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

Autores: 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
Publicado en: Molecular Therapy - Nucleic Acids, Edición 12, 2018, Página(s) 530-542, ISSN 2162-2531
Editor: Nature Publishing Group
DOI: 10.1016/j.omtn.2018.06.010

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

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

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

Autores: Justin S. Antony, Alexander Dewerth, Ashiqul Haque, Rupert Handgretinger, Michael S.D. Kormann
Publicado en: Molecular and Cellular Pediatrics, Edición 2/1, 2015, ISSN 2194-7791
Editor: SpringerOpen
DOI: 10.1186/s40348-015-0022-6

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

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

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

Autores: 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
Publicado en: Nature Biotechnology, 2015, ISSN 1087-0156
Editor: Nature Publishing Group
DOI: 10.1038/nbt.3241

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