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2022
Mark Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector
Liu, Yang ^LU ; Dahl, Maria ^LU ; Debnath, Shubhranshu ^LU ; Rothe, Michael ; Smith, Emma ^LU ; Tan Grahn, Hooi Min ^LU ; Warsi, Sarah ^LU ; Chen, Jun ^LU ; Flygare, Johan ^LU and Schambach, Axel , et al. (2022) In Haematologica 107(2). p.446-456
- Contribution to journal › Article
2021
Mark Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
Dahl, Maria ^LU ; Smith, Emma M.K. ^LU ; Warsi, Sarah ^LU ; Rothe, Michael ; Ferraz, Maria J. ; Aerts, Johannes M.F.G. ; Golipour, Azadeh ; Harper, Claudia ; Pfeifer, Richard and Pizzurro, Daniella , et al. (2021) In Molecular Therapy - Methods and Clinical Development 20. p.312-323
- Contribution to journal › Article
2019
Mark Hematopoietic stem cell-targeted neonatal gene therapy with a clinically applicable lentiviral vector corrects osteopetrosis in oc/oc Mice
Löfvall, Henrik ^LU ; Rothe, Michael ; Schambach, Axel ; Henriksen, Kim ; Richter, Johan ^LU and Moscatelli, Ilana ^LU (2019) In Human Gene Therapy 30(11). p.1395-1404
- Contribution to journal › Article
2018
Mark Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis
Moscatelli, Ilana ^LU ; Löfvall, Henrik ^LU ; Schneider Thudium, Christian ; Rothe, Michael ; Montano, Carmen ^LU ; Kertész, Zsuzsanna ^LU ; Sirin, Mehtap ; Schulz, Ansgar ; Schambach, Axel and Henriksen, Kim , et al. (2018) In Human Gene Therapy 29(8). p.938-949
- Contribution to journal › Article
2017
Mark Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia
Debnath, Shubhranshu ^LU ; Jaako, Pekka ^LU ; Siva, Kavitha ^LU ; Rothe, Michael ; Chen, Jun ^LU ; Dahl, Maria ^LU ; Gaspar, H. Bobby ; Flygare, Johan ^LU ; Schambach, Axel and Karlsson, Stefan ^LU (2017) In Molecular Therapy 25(8). p.1805-1814
- Contribution to journal › Article
2015
Mark Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
Dahl, Maria ^LU ; Doyle, Alexander ^LU ; Olsson, Karin ^LU ; Månsson, Jan-Eric ; Marques, André R A ; Mirzaian, Mina ; Aerts, Johannes M ; Ehinger, Mats ^LU ; Rothe, Michael and Modlich, Ute , et al. (2015) In Molecular Therapy 23(5). p.835-844
- Contribution to journal › Article
2014
Mark Gene therapy cures the anemia and lethal bone marrow failure in mouse model for RPS19-deficient Diamond-Blackfan anemia.
Jaako, Pekka ^LU ; Debnath, Shubhranshu ^LU ; Olsson, Karin ^LU ; Modlich, Ute ; Rothe, Michael ; Schambach, Axel ; Flygare, Johan ^LU and Karlsson, Stefan ^LU (2014) In Haematologica 99(12). p.1792-1798
- Contribution to journal › Article

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