Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy

2010 ◽  
Vol 30 (1) ◽  
pp. 13-18 ◽  
Author(s):  
Sung-Chul Jung ◽  
Eun-Sook Park ◽  
Eun Nam Choi ◽  
Chi Hwa Kim ◽  
Su Jin Kim ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii

The characterization of mouse model of mucopolysaccharidosis type II

2015 ◽  
Vol 114 (2) ◽  
pp. S64
Author(s):  
Masafumi Kinoshita ◽  
Hideto Morimoto ◽  
Eiji Yoden
Keyword(s):  
Mouse Model ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii

HMI-203: Gene therapy developmental candidate for mucopolysaccharidosis type II (MPS II), or Hunter syndrome

2021 ◽  
Vol 132 ◽  
pp. S147
Author(s):  
Kruti Patel ◽  
Laura Smith ◽  
Tania Seabrook ◽  
Alec Tzianabos ◽  
Lindsay Schulman ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Hunter Syndrome ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii ◽  
Mps Ii

scholarly journals Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy

2007 ◽  
Vol 74 (6) ◽  
pp. 429-438 ◽  
Author(s):  
Mayra F. Garcia-Rivera ◽  
Leah E. Colvin-Wanshura ◽  
Matthew S. Nelson ◽  
Zhenhong Nan ◽  
Shaukat A. Khan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Human Stem Cell ◽  
Preclinical Testing ◽  
Cell And Gene Therapy

Blood-brain barrier-penetrating iduronate-2-sulfatase reduces brain glycosaminoglycans in mouse model of mucopolysaccharidosis type II

2018 ◽  
Vol 123 (2) ◽  
pp. S134
Author(s):  
Hiroyuki Sonoda ◽  
Hideto Morimoto ◽  
Eiji Yoden ◽  
Yuri Koshimura ◽  
Masafumi Kinoshita ◽  
...  
Keyword(s):  
Mouse Model ◽  
Blood Brain Barrier ◽  
Brain Barrier ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii ◽  
Blood Brain

scholarly journals Brain RNA-Seq Profiling of the Mucopolysaccharidosis Type II Mouse Model

2017 ◽  
Vol 18 (5) ◽  
pp. 1072 ◽  
Author(s):  
Marika Salvalaio ◽  
Francesca D’Avanzo ◽  
Laura Rigon ◽  
Alessandra Zanetti ◽  
Michela D’Angelo ◽  
...  
Keyword(s):  
Mouse Model ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Rna Seq ◽  
Mucopolysaccharidosis Type Ii

scholarly journals CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)

JCI Insight ◽  
2016 ◽  
Vol 1 (9) ◽  
Author(s):  
Sandra Motas ◽  
Virginia Haurigot ◽  
Miguel Garcia ◽  
Sara Marcó ◽  
Albert Ribera ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Hunter Syndrome ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii

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
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