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

Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

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Vol 132 (2) ◽  
pp. S107
Author(s):  
Niek P. van Til ◽  
Yildirim Dogan ◽  
Cecilia Barese ◽  
Zeenath Unnisa ◽  
Swaroopa Guda ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Preclinical Study ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Long-term follow-up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

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Author(s):  
Pierre BOUGNERES ◽  
Salima Hacein-Bey-Abina ◽  
Ivan Labik ◽  
Catherine ADAMSBAUM ◽  
CLEMENCE CASTAIGNEDE ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Long Term Follow Up ◽  
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

scholarly journals Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE

2018 ◽  
Vol 8 ◽  
pp. 152-165 ◽  
Author(s):  
Rana Yadak ◽  
Raquel Cabrera-Pérez ◽  
Javier Torres-Torronteras ◽  
Marianna Bugiani ◽  
Joost C. Haeck ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Safety Evaluation ◽  
Efficacy And Safety ◽  
Hematopoietic Stem ◽  
Preclinical Efficacy ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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