Development of a stem cell gene therapy for Sanfilippo syndrome type B

2017 ◽  
Vol 120 (1-2) ◽  
pp. S37 ◽  
Author(s):  
Don L Clarke ◽  
Valentina Sanguez ◽  
Shih Kan ◽  
Steven Lee ◽  
Michelina Iacovino ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Stem Cell Gene Therapy for Chronic Renal Failure

2003 ◽  
Vol 3 (5) ◽  
pp. 387-394 ◽  
Author(s):  
T. Yokoo ◽  
K. Sakurai ◽  
T. Ohashi ◽  
T. Kawamura
Keyword(s):  
Gene Therapy ◽  
Renal Failure ◽  
Stem Cell ◽  
Chronic Renal Failure ◽  
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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