Pre-clinical workup of lentiviral mediated stem cell gene therapy for mucopolysaccharidosis type IIIA

2015 ◽  
Vol 114 (2) ◽  
pp. S41
Author(s):  
Stuart M. Ellison ◽  
Ana Sergijenko ◽  
Alex Langford-Smith ◽  
Kia Langford-Smith ◽  
Fiona Wilkinson ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Cell Gene ◽  
Mucopolysaccharidosis Type Iiia ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Lentiviral mediated stem cell gene therapy corrects a mouse model of mucopolysaccharidosis type IIIA

2011 ◽  
Vol 102 (2) ◽  
pp. S8
Author(s):  
Brian Bigger ◽  
Alex Langford-Smith ◽  
Fiona Wilkinson ◽  
Kia Langford-Smith ◽  
Ana Sergijenko ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Cell Gene ◽  
Mucopolysaccharidosis Type Iiia ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II

2015 ◽  
Vol 26 (6) ◽  
pp. 357-366 ◽  
Author(s):  
Taichi Wakabayashi ◽  
Yohta Shimada ◽  
Kazumasa Akiyama ◽  
Takashi Higuchi ◽  
Takahiro Fukuda ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Murine Model ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii ◽  
Hematopoietic Stem ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy

scholarly journals Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms

2018 ◽  
Vol 10 (7) ◽  
Author(s):  
Hélène FE Gleitz ◽  
Ai Yin Liao ◽  
James R Cook ◽  
Samuel F Rowlston ◽  
Gabriella MA Forte ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii ◽  
Cell Gene ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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