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Reduction of heparan sulfate in the brain by pabinafusp alfa results in prevention of neurodegeneration and neurocognitive impairment in a mouse model of mucopolysaccharidosis type II
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2020.12.169
◽
2021
◽
Vol 132
(2)
◽
pp. S73
Author(s):
Hideto Morimoto
◽
Sachiho Kida
◽
Eiji Yoden
◽
Masafumi Kinoshita
◽
Noboru Tanaka
◽
...
Keyword(s):
Mouse Model
◽
Heparan Sulfate
◽
Neurocognitive Impairment
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
◽
The Brain
Download Full-text
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References
Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy
Molecules and Cells
◽
10.1007/s10059-010-0083-2
◽
2010
◽
Vol 30
(1)
◽
pp. 13-18
◽
Cited By ~ 25
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
Download Full-text
Neurologic improvement in a mouse model of Hunter syndrome (mucopolysaccharidosis type II) by treatment with AAV9 vector after the development of cognitive dysfunction
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2016.11.274
◽
2017
◽
Vol 120
(1-2)
◽
pp. S107-S108
Author(s):
Kelly M Podetz-Pedersen
◽
Kanut Laoharawee
◽
Tam T Nguyen
◽
Sajya M Singh
◽
Lucy Vulchanova
◽
...
Keyword(s):
Mouse Model
◽
Cognitive Dysfunction
◽
Hunter Syndrome
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
Download Full-text
Blood-brain barrier-penetrating iduronate-2-sulfatase reduces brain glycosaminoglycans in mouse model of mucopolysaccharidosis type II
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2017.12.368
◽
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
Download Full-text
Brain RNA-Seq Profiling of the Mucopolysaccharidosis Type II Mouse Model
International Journal of Molecular Sciences
◽
10.3390/ijms18051072
◽
2017
◽
Vol 18
(5)
◽
pp. 1072
◽
Cited By ~ 9
Author(s):
Marika Salvalaio
◽
Francesca D’Avanzo
◽
Laura Rigon
◽
Alessandra Zanetti
◽
Michela D’Angelo
◽
...
Keyword(s):
Mouse Model
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Mucopolysaccharidosis Type
◽
Type Ii
◽
Rna Seq
◽
Mucopolysaccharidosis Type Ii
Download Full-text
Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC/MS/MS
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2018.07.013
◽
2018
◽
Vol 125
(1-2)
◽
pp. 53-58
◽
Cited By ~ 11
Author(s):
Noboru Tanaka
◽
Sachiho Kida
◽
Masafumi Kinoshita
◽
Hideto Morimoto
◽
Tadao Shibasaki
◽
...
Keyword(s):
Cerebrospinal Fluid
◽
Heparan Sulfate
◽
Murine Model
◽
High Sensitivity
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
Download Full-text
1024. Electrogene Transfer in Mucopolysaccharidosis Type II Mouse Model Induces High-Level Production of Iduronate-2-Sulfatase in the Skeletal Muscle
Molecular Therapy
◽
10.1016/s1525-0016(16)41466-8
◽
2003
◽
Vol 7
(5)
◽
pp. S395
Keyword(s):
Skeletal Muscle
◽
Mouse Model
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
◽
High Level
◽
Level Production
Download Full-text
Identification of age-dependent motor and neuropsychological behavioural abnormalities in a mouse model of Mucopolysaccharidosis Type II
PLoS ONE
◽
10.1371/journal.pone.0172435
◽
2017
◽
Vol 12
(2)
◽
pp. e0172435
◽
Cited By ~ 10
Author(s):
Hélène F. E. Gleitz
◽
Claire O’Leary
◽
Rebecca J. Holley
◽
Brian W. Bigger
Keyword(s):
Mouse Model
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
◽
Age Dependent
Download Full-text
A commentary on high-dose enzyme replacement therapy attenuates cerebroventriculomegaly in a mouse model of mucopolysaccharidosis type II
Journal of Human Genetics
◽
10.1038/jhg.2013.115
◽
2013
◽
Vol 58
(12)
◽
pp. 767-768
Author(s):
Maria L Escolar
Keyword(s):
Mouse Model
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
High Dose
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
◽
Enzyme Replacement
Download Full-text
Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II
Orphanet Journal of Rare Diseases
◽
10.1186/s13023-015-0356-0
◽
2015
◽
Vol 10
(1)
◽
Cited By ~ 9
Author(s):
Sung Yoon Cho
◽
Jeehun Lee
◽
Ah-Ra Ko
◽
Min Jung Kwak
◽
Sujin Kim
◽
...
Keyword(s):
Mouse Model
◽
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
High Dose
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
◽
Enzyme Replacement
Download Full-text
Distribution of heparan sulfate and dermatan sulfate in mucopolysaccharidosis type II mouse tissues pre- and post-enzyme-replacement therapy determined by UPLC–MS/MS
Bioanalysis
◽
10.4155/bio-2018-0306
◽
2019
◽
Vol 11
(8)
◽
pp. 727-740
◽
Cited By ~ 2
Author(s):
Iskren Menkovic
◽
Pamela Lavoie
◽
Michel Boutin
◽
Christiane Auray-Blais
Keyword(s):
Enzyme Replacement Therapy
◽
Heparan Sulfate
◽
Replacement Therapy
◽
Dermatan Sulfate
◽
Mucopolysaccharidosis Type
◽
Type Ii
◽
Mucopolysaccharidosis Type Ii
◽
Enzyme Replacement
◽
Mouse Tissues
Download Full-text
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