scholarly journals In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

2018 ◽  
Vol 129 (2) ◽  
pp. 598-615 ◽  
Author(s):  
Hongjie Wang ◽  
Aphrodite Georgakopoulou ◽  
Nikoletta Psatha ◽  
Chang Li ◽  
Chrysi Capsali ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Thalassemia Intermedia ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements

JCI Insight ◽  
2020 ◽  
Vol 5 (16) ◽  
Author(s):  
Hongjie Wang ◽  
Aphrodite Georgakopoulou ◽  
Chang Li ◽  
Zhinan Liu ◽  
Sucheol Gil ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Hematopoietic Stem Cell ◽  
Regulatory Elements ◽  
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
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