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Personalized Nanomedicines for Leukemia Patients

Total Cost €


EC-Contrib. €






Project "PNANOMED" data sheet

The following table provides information about the project.


Organization address
address: Carl-Neuberg-Strasse 1
postcode: 30625

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]
 Total cost 1˙499˙750 €
 EC max contribution 1˙499˙750 € (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 2015-07-01   to  2020-06-30


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 


 Project objective

Acute myeloid leukemia (AML) remains a devastating disease, while progress in genetic characterization and nanomedical approaches promise a new era of individualized treatments. To prioritize genetic aberrations in AML for therapeutic targeting and to develop a pipeline for personalized nanomedicines I will i) establish a biobank of transplantable primary human AML xenotransplants, ii) fully characterize the genetic landscape of these leukemias, iii) establish the functional hierarchy of driver and passenger mutations in these human leukemia models, iv) develop highly efficient nanoparticle-siRNA formulations that allow in vivo delivery of siRNA to primary AML blasts, and v) design double specific siRNA-based nanomedicines for improved efficacy and tolerability. The expertise of my research team and my institutional settings and collaborations provide a unique platform to achieve these objectives. My access to freshly isolated leukemia blasts allows efficient establishment of a biobank for AML xenotransplant models. In fact, we can serially transplant and expand primary AML cells in immunodeficient mice. The biobank will be an invaluable resource for pharmaceutical product development. I have extensive experience in the genetic characterization and functional evaluation of leukemic cells, which I will apply to the newly generated human AML models. I will use inducible lentiviral approaches to genetically modify human leukemia cells and observe the functional effects in vivo, to identify the relevant targets for leukemogenicity of each primary AML model. Most importantly, I can formulate nanoparticle-siRNA systems that show unprecedented complete uptake into human leukemia cells in vivo and open the door for specific inhibition of any gene. These established tools provide me with the unique ability to develop a pipeline for individualized nanomedicines that will improve AML treatment and will also have broad applications beyond leukemia treatment.


year authors and title journal last update
List of publications.
2017 Nicolaus Kröger, Victoria Panagiota, Anita Badbaran, Tatjana Zabelina, Ioanna Triviai, Michelle Maria Araujo Cruz, Rabia Shahswar, Francis Ayuk, Marten Gehlhaar, Christine Wolschke, Robin Bollin, Carolin Walter, Martin Dugas, Lutz Wiehlmann, Ulrich Lehmann, Christian Koenecke, Anuhar Chaturvedi, Haefaa Alchalby, Michael Stadler, Matthias Eder, Max Christopeit, Gudrun Göhring, Michael Koenigsmann, Brigitte Schlegelberger, Hans-Heinrich Kreipe, Arnold Ganser, Carol Stocking, Boris Fehse, Felicitas Thol, Michael Heuser
Impact of Molecular Genetics on Outcome in Myelofibrosis Patients after Allogeneic Stem Cell Transplantation
published pages: 1095-1101, ISSN: 1083-8791, DOI: 10.1016/j.bbmt.2017.03.034
Biology of Blood and Marrow Transplantation 23/7 2019-05-29
2017 N Jyotsana, A Sharma, A Chaturvedi, M Scherr, F Kuchenbauer, L Sajti, A Barchanski, R Lindner, F Noyan, K-W Sühs, D Grote-Koska, K Brand, H-P Vornlocher, M Stanulla, B Bornhauser, J-P Bourquin, M Eder, F Thol, A Ganser, R K Humphries, E Ramsay, P Cullis, M Heuser
RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo
published pages: 224-226, ISSN: 0887-6924, DOI: 10.1038/leu.2017.269
Leukemia 32/1 2019-05-29
2017 Nidhi Jyotsana, Michael Heuser
Exploiting differential RNA splicing patterns: a potential new group of therapeutic targets in cancer
published pages: 107-121, ISSN: 1472-8222, DOI: 10.1080/14728222.2018.1417390
Expert Opinion on Therapeutic Targets 22/2 2019-05-29
2017 F Thol, S Klesse, L Köhler, R Gabdoulline, A Kloos, A Liebich, M Wichmann, A Chaturvedi, J Fabisch, V I Gaidzik, P Paschka, L Bullinger, G Bug, H Serve, G Göhring, B Schlegelberger, M Lübbert, H Kirchner, M Wattad, D Kraemer, B Hertenstein, G Heil, W Fiedler, J Krauter, R F Schlenk, K Döhner, H Döhner, A Ganser, M Heuser
Acute myeloid leukemia derived from lympho-myeloid clonal hematopoiesis
published pages: 1286-1295, ISSN: 0887-6924, DOI: 10.1038/leu.2016.345
Leukemia 31/6 2019-05-29
2017 Michael Heuser, Razif Gabdoulline, Patrick Löffeld, Vera Dobbernack, Henriette Kreimeyer, Mira Pankratz, Madita Flintrop, Alessandro Liebich, Sabrina Klesse, Victoria Panagiota, Michael Stadler, Martin Wichmann, Rabia Shahswar, Uwe Platzbecker, Christian Thiede, Thomas Schroeder, Guido Kobbe, Robert Geffers, Brigitte Schlegelberger, Gudrun Göhring, Hans-Heinrich Kreipe, Ulrich Germing, Arnold Ganser, Nicolaus Kröger, Christian Koenecke, Felicitas Thol
Individual outcome prediction for myelodysplastic syndrome (MDS) and secondary acute myeloid leukemia from MDS after allogeneic hematopoietic cell transplantation
published pages: 1361-1372, ISSN: 0939-5555, DOI: 10.1007/s00277-017-3027-5
Annals of Hematology 96/8 2019-05-29
2016 A Chaturvedi, M M Araujo Cruz, N Jyotsana, A Sharma, R Goparaju, A Schwarzer, K Görlich, R Schottmann, E A Struys, E E Jansen, C Rohde, C Müller-Tidow, R Geffers, G Göhring, A Ganser, F Thol, M Heuser
Enantiomer-specific and paracrine leukemogenicity of mutant IDH metabolite 2-hydroxyglutarate
published pages: 1708-1715, ISSN: 0887-6924, DOI: 10.1038/leu.2016.71
Leukemia 30/8 2019-05-29
2016 M. Heuser
Therapy-related myeloid neoplasms: does knowing the origin help to guide treatment?
published pages: 24-32, ISSN: 1520-4391, DOI: 10.1182/asheducation-2016.1.24
Hematology 2016/1 2019-05-29
2017 A Chaturvedi, L Herbst, S Pusch, L Klett, R Goparaju, D Stichel, S Kaulfuss, O Panknin, K Zimmermann, L Toschi, R Neuhaus, A Haegebarth, H Rehwinkel, H Hess-Stumpp, M Bauser, T Bochtler, E A Struys, A Sharma, A Bakkali, R Geffers, M M Araujo-Cruz, F Thol, R Gabdoulline, A Ganser, A D Ho, A von Deimling, K Rippe, M Heuser, A Krämer
Pan-mutant-IDH1 inhibitor BAY1436032 is highly effective against human IDH1 mutant acute myeloid leukemia in vivo
published pages: 2020-2028, ISSN: 0887-6924, DOI: 10.1038/leu.2017.46
Leukemia 31/10 2019-05-29
2017 Michael Heuser, Haiyang Yun, Felicitas Thol
Epigenetics in myelodysplastic syndromes
published pages: , ISSN: 1044-579X, DOI: 10.1016/j.semcancer.2017.07.009
Seminars in Cancer Biology 2019-05-29

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