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

Total Cost €


EC-Contrib. €






 BREATHE project word cloud

Explore the words cloud of the BREATHE project. It provides you a very rough idea of what is the project "BREATHE" about.

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Project "BREATHE" data sheet

The following table provides information about the project.


Organization address
postcode: 72074

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country Germany [DE]
 Project website
 Total cost 1˙497˙125 €
 EC max contribution 1˙497˙125 € (100%)
 Programme 1. H2020-EU.1.1. (EXCELLENT SCIENCE - European Research Council (ERC))
 Code Call ERC-2014-STG
 Funding Scheme ERC-STG
 Starting year 2015
 Duration (year-month-day) from 2015-04-01   to  2020-03-31


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 


 Project objective

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
List of publications.
2018 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
2019 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
2018 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
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
2018 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
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
2015 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
2016 Franziska Zeyer, Benedikt Mothes, Clara Will, Melanie Carevic, Jennifer Rottenberger, Bernd Nürnberg, 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
2016 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:
Discovery Medicine 2019-05-29

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