scholarly journals 783. Correction of βc-Deficient Pulmonary Alveolar Proteinosis (PAP) by Hematopoietic Stem Cell Gene Transfer in a Murine Model

2006 ◽  
Vol 13 ◽  
pp. S303
Author(s):  
V. Kleff ◽  
U.R. Sorg ◽  
C. Bury ◽  
B. Opalka ◽  
M. Flasshove ◽  
...  
Keyword(s):  
Stem Cell ◽  
Gene Transfer ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Pulmonary Alveolar Proteinosis ◽  
Hematopoietic Stem ◽  
Alveolar Proteinosis ◽  
Cell Gene ◽  
Stem Cell Gene

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

Haematologica ◽  
2019 ◽  
Vol 105 (4) ◽  
pp. 1147-1157
Author(s):  
Miriam Hetzel ◽  
Elena Lopez-Rodriguez ◽  
Adele Mucci ◽  
Ariane Hai Ha Nguyen ◽  
Takuji Suzuki ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Pulmonary Alveolar Proteinosis ◽  
Hematopoietic Stem ◽  
Alveolar Proteinosis

Long-term efficacy and safety of hematopoietic stem cell gene therapy mediated by lentiviral vectors in the murine model of Wiskott-Aldrich syndrome

2008 ◽  
Vol 40 (2) ◽  
pp. 256-257
Author(s):  
Marita Bosticardo ◽  
Francesco Marangoni ◽  
Michela Locci ◽  
Elena Draghici ◽  
Samantha Scaramuzza ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease

2020 ◽  
Vol 18 ◽  
pp. 558-570
Author(s):  
Giuseppa Piras ◽  
Claudia Montiel-Equihua ◽  
Yee-Ka Agnes Chan ◽  
Slawomir Wantuch ◽  
Daniel Stuckey ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Murine Model ◽  
Pompe Disease ◽  
Hematopoietic Stem ◽  
Clinical Phenotypes ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

2006 ◽  
Vol 24 (6) ◽  
pp. 687-696 ◽  
Author(s):  
Eugenio Montini ◽  
Daniela Cesana ◽  
Manfred Schmidt ◽  
Francesca Sanvito ◽  
Maurilio Ponzoni ◽  
...  
Keyword(s):  
Stem Cell ◽  
Gene Transfer ◽  
Mouse Model ◽  
Hematopoietic Stem Cell ◽  
Lentiviral Vector ◽  
Hematopoietic Stem ◽  
Vector Integration ◽  
Cell Gene ◽  
Stem Cell Gene
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