scholarly journals Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation

2020 ◽  
Vol 19 ◽  
pp. 261-274
Author(s):  
Miho Wada ◽  
Yohta Shimada ◽  
Sayoko Iizuka ◽  
Natsumi Ishii ◽  
Hiromi Hiraki ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Bone Remodeling ◽  
Mouse Models ◽  
Ex Vivo ◽  
Mucopolysaccharidosis Type ◽  
Type Ii ◽  
Mucopolysaccharidosis Type Ii ◽  
Ex Vivo Gene Therapy ◽  
Bone Complications

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

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

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

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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