scholarly journals Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

2018 ◽  
Vol 53 (7) ◽  
pp. 913-917 ◽  
Author(s):  
Valeria Calbi ◽  
Francesca Fumagalli ◽  
Giulia Consiglieri ◽  
Rachele Penati ◽  
Serena Acquati ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Metachromatic Leukodystrophy ◽  
Endothelial Injury ◽  
Hematopoietic Stem ◽  
Post Transplant ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

A Mutation-Agnostic Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy

2021 ◽  
Author(s):  
Justin S. Antony ◽  
Alberto Daniel-Moreno ◽  
Andrés Lamsfus-Calle ◽  
Janani Raju ◽  
Merve Kaftancioglu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Metachromatic Leukodystrophy ◽  
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

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 Development of a modified selective amplifier gene for hematopoietic stem cell gene therapy

Gene Therapy ◽  
1999 ◽  
Vol 6 (6) ◽  
pp. 1038-1044 ◽  
Author(s):  
K M Matsuda ◽  
A Kume ◽  
Y Ueda ◽  
M Urabe ◽  
M Hasegawa ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Selective Amplifier ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Promises and Challenges in Hematopoietic Stem Cell Gene Therapy

2017 ◽  
Vol 28 (10) ◽  
pp. 782-799 ◽  
Author(s):  
Saskia Kohlscheen ◽  
Halvard Bonig ◽  
Ute Modlich
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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