Explore the words cloud of the BREATHE project. It provides you a very rough idea of what is the project "BREATHE" about.
The following table provides information about the project.
EBERHARD KARLS UNIVERSITAET TUEBINGEN
|Coordinator Country||Germany [DE]|
|Total cost||1˙497˙125 €|
|EC max contribution||1˙497˙125 € (100%)|
1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
|Duration (year-month-day)||from 2015-04-01 to 2020-03-31|
Take a look of project's partnership.
|1||EBERHARD KARLS UNIVERSITAET TUEBINGEN||DE (TUEBINGEN)||coordinator||1˙497˙125.00|
Surfactant Protein B (SP-B) deficiency and Cystic Fibrosis (CF) are severe, fatal inherited diseases affecting the lungs of ten thousands of people, for which there is currently no available cure. Although gene therapy is a promising therapeutic approach, various technical problems, including numerous physical and immune-mediated barriers, have prevented successful application to date. My recent studies were the first to demonstrate the life-saving efficacy of repeated pulmonary delivery of chemically modified messenger RNA (mRNA) in a mouse model of congenital SP-B deficiency. By incorporating balanced amounts of modified nucleotides to mimic endogenous transcripts, I developed a safe and therapeutically efficient vehicle for lung transfection that eliminates the risk of genomic integration commonly associated with DNA-based vectors. I also assessed the delivery of mRNA-encoded site-specific nucleases to the lung to facilitate targeted gene correction of the underlying disease-causing mutations. In comprehensive studies, we show that a single application of nucleases encoded by nucleotide-modified RNA (nec-mRNA) can generate in vivo correction of terminally differentiated alveolar type II cells, which more than quadrupled the life span of SP-B deficient mice. Together with my working group, I aim to further develop this technology to enhance the efficiency and safety of nec-mRNA-mediated in vivo lung stem cell targeting, providing an ultimate cure by permanent correction. Specifically, we will test this approach in humanized mouse models of SP-B deficiency and CF. Developing and genetically engineering humanized models in vivo will be a critical step towards the safe translation of mRNA based nuclease technology to the clinic. With my competitive edge in lung-transfection technology and strong data, I feel that my group is uniquely suited to achieve these goals and to make a highly valuable contribution to the development of an efficient treatment.
|year||authors and title||journal||last update|
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
Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification
published pages: 530-542, ISSN: 2162-2531, DOI: 10.1016/j.omtn.2018.06.010
|Molecular Therapy - Nucleic Acids 12||2019-12-17|
Itishri Sahu, A.K.M. Ashiqul Haque, Brian Weidensee, Petra Weinmann, Michael S.D. Kormann
Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases
published pages: 803-823, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2019.02.019
|Molecular Therapy 27/4||2019-12-17|
Justin S. Antony, Ngadhnjim Latifi, A. K. M. Ashiqul Haque, AndrÃ©s Lamsfus-Calle, Alberto Daniel-Moreno, Sebastian Graeter, Praveen Baskaran, Petra Weinmann, Markus Mezger, Rupert Handgretinger, Michael S. D. Kormann
Gene correction of HBB mutations in CD34+ hematopoietic stem cells using Cas9 mRNA and ssODN donors
published pages: , ISSN: 2194-7791, DOI: 10.1186/s40348-018-0086-1
|Molecular and Cellular Pediatrics 5/1||2019-12-17|
A. K. M. Ashiqul Haque, Alexander Dewerth, Justin S. Antony, Joachim RiethmÃ¼ller, 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
Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis
published pages: , ISSN: 2045-2322, DOI: 10.1038/s41598-018-34960-0
|Scientific Reports 8/1||2019-12-17|
Justin S. Antony, Alexander Dewerth, Ashiqul Haque, Rupert Handgretinger, Michael S.D. Kormann
Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies
published pages: , ISSN: 2194-7791, DOI: 10.1186/s40348-015-0022-6
|Molecular and Cellular Pediatrics 2/1||2019-05-29|
Franziska Zeyer, Benedikt Mothes, Clara Will, Melanie Carevic, Jennifer Rottenberger, Bernd NÃ¼rnberg, Dominik Hartl, Rupert Handgretinger, Sandra Beer-Hammer, Michael S. D. Kormann
mRNA-Mediated Gene Supplementation of Toll-Like Receptors as Treatment Strategy for Asthma In Vivo
published pages: e0154001, ISSN: 1932-6203, DOI: 10.1371/journal.pone.0154001
|PLOS ONE 11/4||2019-05-29|
Satheesh Elangovan,corresponding author1 Michael S.D. Kormann,2 Behnoush Khorsand,3 and Aliasger K. Salemcorresponding author1,3
The Oral and Craniofacial Relevance of Chemically Modified RNA Therapeutics
published pages: , ISSN: 1539-6509, DOI:
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