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- 2021
- Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
- 2019
-
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
Mark
- 2018
-
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
Mark
- 2017
-
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
Mark
- 2015
- Lentiviral gene therapy using cellular promoters cures type 1 Gaucher disease in mice.
- 2014
-
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
Mark