iPS-derived neural stem cells for disease modeling and evaluation of therapeutics for mucopolysaccharidosis type II

Type Ii ◽

Mucopolysaccharidosis Type Ii

Neural cells generated from human induced pluripotent stem cells as a model of CNS involvement in mucopolysaccharidosis type II

Journal of Inherited Metabolic Disease ◽

10.1007/s10545-017-0108-5 ◽

2017 ◽

Vol 41 (2) ◽

pp. 221-229 ◽

Cited By ~ 5

Author(s):

Jitka Rybová ◽

Jana Ledvinová ◽

Jakub Sikora ◽

Ladislav Kuchař ◽

Robert Dobrovolný

Keyword(s):

Stem Cells ◽

Induced Pluripotent Stem Cells ◽

Pluripotent Stem Cells ◽

Neural Cells ◽

Type Ii ◽

Cns Involvement ◽

Induced Pluripotent

495. Prevention of Neurocognitive Deficit by Ex Vivo Lentiviral Transduction of Hematopoietic Stem Cells in a Murine Model of Mucopolysaccharidosis Type II

Molecular Therapy ◽

10.1016/s1525-0016(16)34830-4 ◽

2013 ◽

Vol 21 ◽

pp. S191 ◽

Cited By ~ 1

Keyword(s):

Stem Cells ◽

Hematopoietic Stem Cells ◽

Murine Model ◽

Ex Vivo ◽

Type Ii ◽

Hematopoietic Stem ◽

Lentiviral Transduction ◽

Neurocognitive Deficit

86. Ex vivo lentiviral gene transfer targeting hematopoietic stem cells for therapy of mucopolysaccharidosis type II, Hunter syndrome

10.1016/j.ymgme.2008.11.087 ◽

2009 ◽

Vol 96 (2) ◽

pp. S30-S31

Author(s):

R. Scott McIvor ◽

Daniel A. Wolf ◽

Kelly Podetz-Petersen ◽

Zhenhong Nan ◽

Chester B. Whitley ◽

...

Keyword(s):

Stem Cells ◽

Gene Transfer ◽

Hematopoietic Stem Cells ◽

Ex Vivo ◽

Hunter Syndrome ◽

Type Ii ◽

Hematopoietic Stem

Ex vivo lentiviral transduction of hematopoietic stem cells in mucopolysaccharidosis type II (MPS II) mice achieves high levels of systemic iduronate-2-sulfatase (IDS) enzyme activity and normalization of glycosaminoglycans (GAGs)

10.1016/j.ymgme.2020.12.247 ◽

2021 ◽

Vol 132 (2) ◽

pp. S102

Author(s):

Miles C. Smith ◽

Lalitha Belur ◽

Olivia Erlanson ◽

Kelly Podetz-Pedersen ◽

Andrea Karlen ◽

...

Keyword(s):

Stem Cells ◽

Enzyme Activity ◽

Hematopoietic Stem Cells ◽

Ex Vivo ◽

Type Ii ◽

Hematopoietic Stem ◽

Lentiviral Transduction ◽

Mps Ii

Orthopedic manifestations in patients with mucopolysaccharidosis type II enrolled in the Hunter Outcome Survey

10.1016/j.ymgme.2020.12.148 ◽

2021 ◽

Vol 132 (2) ◽

pp. S65

Author(s):

Bianca Link ◽

Jaco Botha ◽

Roberto Giugliani

Keyword(s):

Type Ii ◽

Mucopolysaccharidosis Type Ii

Divergent developmental trajectories in two siblings with neuropathic mucopolysaccharidosis type II (Hunter syndrome) receiving conventional and novel enzyme replacement therapies: A case report

JIMD Reports ◽

10.1002/jmd2.12239 ◽

2021 ◽

Author(s):

Kazuyoshi Tomita ◽

Shungo Okamoto ◽

Toshiyuki Seto ◽

Takashi Hamazaki ◽

Sairei So ◽

...

Keyword(s):

Case Report ◽

Developmental Trajectories ◽

Hunter Syndrome ◽

Type Ii ◽

Enzyme Replacement

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

10.1016/s1096-7192(21)00315-2 ◽

2021 ◽

Vol 132 ◽

pp. S147

Author(s):

Kruti Patel ◽

Laura Smith ◽

Tania Seabrook ◽

Alec Tzianabos ◽

Lindsay Schulman ◽

...

Keyword(s):

Gene Therapy ◽

Hunter Syndrome ◽

Type Ii ◽

Mps Ii

Comparison of cognitive function in siblings with neuronopathic mucopolysaccharidosis type II: Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT

10.1016/j.ymgme.2020.12.170 ◽

2021 ◽

Vol 132 (2) ◽

pp. S73

Author(s):

Joseph Muenzer ◽

Barbara K. Burton ◽

Paul Harmatz ◽

Luis González Gutiérrez-Solana ◽

Matilde Ruiz-Garcia ◽

...

Keyword(s):

Cognitive Function ◽

Early Treatment ◽

Type Ii ◽

Mucopolysaccharidosis Type Ii

Generation of Mucopolysaccharidosis type II (MPS II) human induced pluripotent stem cell (iPSC) line from a 1-year-old male with pathogenic IDS mutation

Stem Cell Research ◽

10.1016/j.scr.2016.09.033 ◽

2016 ◽

Vol 17 (3) ◽

pp. 482-484 ◽

Cited By ~ 4

Author(s):

Eszter Varga ◽

Csilla Nemes ◽

István Bock ◽

Norbert Varga ◽

Anita Fehér ◽

...

Keyword(s):

Stem Cell ◽

Pluripotent Stem Cell ◽

Induced Pluripotent Stem Cell ◽

Type Ii ◽

Ipsc Line ◽

Mps Ii ◽

Induced Pluripotent

Successful reduction of high-sustained anti-idursulfase antibody titers by immune modulation therapy in a patient with severe mucopolysaccharidosis type II

Molecular Genetics and Metabolism Reports ◽

10.1016/j.ymgmr.2014.11.007 ◽

2015 ◽

Vol 2 ◽

pp. 20-24 ◽

Cited By ~ 6

Author(s):

Katherine H. Kim ◽

Yoav H. Messinger ◽

Barbara K. Burton

Keyword(s):

Immune Modulation ◽

Type Ii ◽

Antibody Titers ◽

Successful Reduction