Human Lentiviral Gene Therapy Restores the Cellular Phenotype of Autosomal Recessive Complete IFN-γR1 Deficiency

por Hahn, Katharina, Pollmann, Liart, Nowak, Juliette, Nguyen, Ariane Hai Ha, Haake, Kathrin, Neehus, Anna-Lena, Waqas, Syed F. Hassnain, Pessler, Frank, Baumann, Ulrich, Hetzel, Miriam, Casanova, Jean-Laurent, Schulz, Ansgar, Bustamante, Jacinta, Ackermann, Mania, Lachmann, Nico
Publicado 2020

ISG15 deficiency features a complex cellular phenotype that responds to treatment with itaconate and derivatives

por Waqas, Syed Fakhar‐ul‐Hassnain, Sohail, Aaqib, Nguyen, Ariane Hai Ha, Usman, Abdulai, Ludwig, Tobias, Wegner, Andre, Malik, Muhammad Nasir Hayat, Schuchardt, Sven, Geffers, Robert, Winterhoff, Moritz, Merkert, Sylvia, Martin, Ulrich, Olmer, Ruth, Lachmann, Nico, Pessler, Frank
Publicado 2022

Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis

por Hetzel, Miriam, Lopez-Rodriguez, Elena, Mucci, Adele, Nguyen, Ariane Hai Ha, Suzuki, Takuji, Shima, Kenjiro, Buchegger, Theresa, Dettmer, Sabine, Rodt, Thomas, Bankstahl, Jens P., Malik, Punam, Knudsen, Lars, Schambach, Axel, Hansen, Gesine, Trapnell, Bruce C., Lachmann, Nico, Moritz, Thomas
Publicado 2020