scholarly journals Single-Cell Mapping of Human Hematopoietic Stem/Progenitor Cells States and Ex Vivo Dynamics: A Next Generation Approach to Stem Cell Gene Therapy Drug Product Characterization

2021 ◽  
Vol 27 (3) ◽  
pp. S34-S35
Author(s):  
Cristina Baricordi ◽  
Hareesh Chamarthi ◽  
Mariana Loperfido ◽  
Aimin Yan ◽  
Luigi Barbarossa ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Ex Vivo ◽  
Drug Product ◽  
Cell Mapping ◽  
Hematopoietic Stem ◽  
Product Characterization ◽  
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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