scholarly journals Curative therapy for hemoglobinopathies: an International Society for Cell & Gene Therapy Stem Cell Engineering Committee review comparing outcomes, accessibility and cost of ex vivo stem cell gene therapy versus allogeneic hematopoietic stem cell transplantation

Cytotherapy ◽  
2021 ◽  
Author(s):  
Alexis Leonard ◽  
Alice Bertaina ◽  
Carmem Bonfim ◽  
Sandra Cohen ◽  
Susan Prockop ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Ex Vivo ◽  
Cell Engineering ◽  
Hematopoietic Stem ◽  
Curative Therapy ◽  
Stem Cell Engineering ◽  
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

2021 ◽  
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

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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