Mucopolysaccharidosis type II: one hundred years of research, diagnosis, and treatment
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) was first described by Dr. Charles
Hunter in 1917. Since then, about one hundred years have passed and Hunter syndrome …
Hunter in 1917. Since then, about one hundred years have passed and Hunter syndrome …
Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice
JC Ullman, A Arguello, JA Getz, A Bhalla… - Science Translational …, 2020 - science.org
Most lysosomal storage diseases (LSDs) involve progressive central nervous system (CNS)
impairment, resulting from deficiency of a lysosomal enzyme. Treatment of neuronopathic …
impairment, resulting from deficiency of a lysosomal enzyme. Treatment of neuronopathic …
Presentation and treatments for mucopolysaccharidosis type II (MPS II; Hunter syndrome)
M Stapleton, F Kubaski, RW Mason… - Expert opinion on …, 2017 - Taylor & Francis
ABSTRACT Introduction: Mucopolysaccharidosis Type II (MPS II; Hunter syndrome) is an X-
linked lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS). IDS …
linked lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase (IDS). IDS …
Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I
E Kakkis, M McEntee, C Vogler, S Le, B Levy… - Molecular genetics and …, 2004 - Elsevier
Enzyme replacement therapy (ERT) has been developed for several lysosomal storage
disorders, including mucopolysaccharidosis I (MPS I), and is effective at reducing lysosomal …
disorders, including mucopolysaccharidosis I (MPS I), and is effective at reducing lysosomal …
A molecular genetics view on Mucopolysaccharidosis Type II
S Verma, S Pantoom, J Petters, AK Pandey… - … Research/Reviews in …, 2021 - Elsevier
Abstract Mucopolysaccharidosis Type II (MPS II) is an X-linked recessive genetic disorder
that primarily affects male patients. With an incidence of 1 in 100,000 male live births, the …
that primarily affects male patients. With an incidence of 1 in 100,000 male live births, the …
The biosynthesis and catabolism of galactosaminoglycans
V Prabhakar, R Sasisekharan - Advances in Pharmacology, 2006 - Elsevier
Publisher Summary This chapter discusses the structure of galactosaminoglycans (GalAGs),
their roles in biology and medicine, GalAG biosynthesis, their in vivo cellular degradation …
their roles in biology and medicine, GalAG biosynthesis, their in vivo cellular degradation …
Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy
SC Jung, ES Park, EN Choi, CH Kim, SJ Kim, DK Jin - Molecules and cells, 2010 - Springer
Abstract Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is an X-linked inherited
disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS), which results in …
disorder caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS), which results in …
Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients
PI Dickson, AH Chen - Current pharmaceutical biotechnology, 2011 - ingentaconnect.com
Intrathecal enzyme replacement therapy has been proposed to treat central nervous system
(CNS) disease due to mucopolysaccharidosis type I. Our research has shown that repeated …
(CNS) disease due to mucopolysaccharidosis type I. Our research has shown that repeated …
Fragments of p97 and uses thereof
TZ Vitalis, R Gabathuler - US Patent 9,364,567, 2016 - Google Patents
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[C@@](O)(CC= 2C (O)= C3C (= O) C= 4C= CC= C (C= 4C (= O) C3= C (O) C= 21) OC) C …
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Reduction of GAG storage in MPS II mouse model following implantation of encapsulated recombinant myoblasts
A Friso, R Tomanin, S Alba… - The Journal of Gene …, 2005 - Wiley Online Library
Background Hunter syndrome, mucopolysaccharidosis type II (MPS II), is a X‐linked
inherited disorder caused by the deficiency of the enzyme iduronate‐2‐sulfatase (IDS) …
inherited disorder caused by the deficiency of the enzyme iduronate‐2‐sulfatase (IDS) …