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

por Antony, Justin S., Dewerth, Alexander, Haque, Ashiqul, Handgretinger, Rupert, Kormann, Michael S.D.
Publicado 2015

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

por Kormann, Michael S. D., Dewerth, Alexander, Eichner, Felizitas, Baskaran, Praveen, Hector, Andreas, Regamey, Nicolas, Hartl, Dominik, Handgretinger, Rupert, Antony, Justin S.
Publicado 2017

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

por Zeyer, Franziska, Mothes, Benedikt, Will, Clara, Carevic, Melanie, Rottenberger, Jennifer, Nürnberg, Bernd, Hartl, Dominik, Handgretinger, Rupert, Beer-Hammer, Sandra, Kormann, Michael S. D.
Publicado 2016

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

por Zeyer, Franziska, Mothes, Benedikt, Will, Clara, Carevic, Melanie, Rottenberger, Jennifer, Nürnberg, Bernd, Hartl, Dominik, Handgretinger, Rupert, Beer-Hammer, Sandra, Kormann, Michael S. D.
Publicado 2018

Gene correction of HBB mutations in CD34(+) hematopoietic stem cells using Cas9 mRNA and ssODN donors

por Antony, Justin S., Latifi, Ngadhnjim, Haque, A. K. M. Ashiqul, Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Graeter, Sebastian, Baskaran, Praveen, Weinmann, Petra, Mezger, Markus, Handgretinger, Rupert, Kormann, Michael S. D.
Publicado 2018

Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34(+) HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin

por Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Antony, Justin S., Epting, Thomas, Heumos, Lukas, Baskaran, Praveen, Admard, Jakob, Casadei, Nicolas, Latifi, Ngadhnjim, Siegmund, Darina M., Kormann, Michael S. D., Handgretinger, Rupert, Mezger, Markus
Publicado 2020

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

por Vaidyanathan, Sriram, Azizian, Krist T., Haque, A.K.M. Ashiqul, Henderson, Jordana M., Hendel, Ayal, Shore, Sabrina, Antony, Justin S., Hogrefe, Richard I., Kormann, Michael S.D., Porteus, Matthew H., McCaffrey, Anton P.
Publicado 2018

The Chitinase-Like Protein YKL-40 Modulates Cystic Fibrosis Lung Disease

por Hector, Andreas, Kormann, Michael S. D., Mack, Ines, Latzin, Philipp, Casaulta, Carmen, Kieninger, Elisabeth, Zhou, Zhe, Yildirim, Ali Ö., Bohla, Alexander, Rieber, Nikolaus, Kappler, Matthias, Koller, Barbara, Eber, Ernst, Eickmeier, Olaf, Zielen, Stefan, Eickelberg, Oliver, Griese, Matthias, Mall, Marcus A., Hartl, Dominik
Publicado 2011

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

por Haque, A. K. M. Ashiqul, Dewerth, Alexander, Antony, Justin S., Riethmüller, Joachim, Schweizer, Georg R., Weinmann, Petra, Latifi, Ngadhnjim, Yasar, Hanzey, Pedemonte, Nicoletta, Sondo, Elvira, Weidensee, Brian, Ralhan, Anjali, Laval, Julie, Schlegel, Patrick, Seitz, Christian, Loretz, Brigitta, Lehr, Claus-Michael, Handgretinger, Rupert, Kormann, Michael S. D.
Publicado 2018

Human pluripotent stem cell-derived acinar/ductal organoids generate human pancreas upon orthotopic transplantation and allow disease modelling

por Hohwieler, Meike, Illing, Anett, Hermann, Patrick C, Mayer, Tobias, Stockmann, Marianne, Perkhofer, Lukas, Eiseler, Tim, Antony, Justin S, Müller, Martin, Renz, Susanne, Kuo, Chao-Chung, Lin, Qiong, Sendler, Matthias, Breunig, Markus, Kleiderman, Susanne M, Lechel, André, Zenker, Martin, Leichsenring, Michael, Rosendahl, Jonas, Zenke, Martin, Sainz, Bruno, Mayerle, Julia, Costa, Ivan G, Seufferlein, Thomas, Kormann, Michael, Wagner, Martin, Liebau, Stefan, Kleger, Alexander
Publicado 2017