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561. Expansion of FoxP3+ Regulatory T Cells in Mucopolysaccharidosis Type I Mice Prior to Lentiviral Gene Transfer Promotes Tolerance to α-l-Iduronidase
Molecular Therapy
◽
10.1016/s1525-0016(16)36365-1
◽
2012
◽
Vol 20
◽
pp. S217
Keyword(s):
T Cells
◽
Gene Transfer
◽
Regulatory T Cells
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
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References
Improved gene transfer and normalized enzyme levels in primitive hematopoietic progenitors from patients with mucopolysaccharidosis type I using a bioreactor
The Journal of Gene Medicine
◽
10.1002/jgm.621
◽
2004
◽
Vol 6
(12)
◽
pp. 1293-1303
◽
Cited By ~ 4
Author(s):
Dao Pan
◽
David F. Stroncek
◽
Chester B. Whitley
Keyword(s):
Gene Transfer
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Hematopoietic Progenitors
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Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
Neurobiology of Disease
◽
10.1016/j.nbd.2011.02.015
◽
2011
◽
Vol 43
(1)
◽
pp. 123-133
◽
Cited By ~ 48
Author(s):
Daniel A. Wolf
◽
Andrew W. Lenander
◽
Zhenhong Nan
◽
Lalitha R. Belur
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◽
...
Keyword(s):
Gene Transfer
◽
Murine Model
◽
Direct Gene Transfer
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Neurologic Disease
◽
Mucopolysaccharidosis Type I
◽
Direct Gene
Download Full-text
Long-term restoration of alpha-L-iduronidase activity in fibroblasts from patients with mucopolysaccharidosis type I after non-viral gene transfer
Clinical & Biomedical Research
◽
10.4322/2357-9730.74048
◽
2017
◽
Vol 37
(4)
◽
pp. 330-333
Author(s):
Fabiano de Oliveira Poswar
◽
Fabiana Quoos Mayer
◽
Maira Burin
◽
Ursula da Silveira Matte
◽
Roberto Giugliani
◽
...
Keyword(s):
Gene Transfer
◽
Viral Gene
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Viral Gene Transfer
Download Full-text
Faculty Opinions recommendation of Type I interferons directly inhibit regulatory T cells to allow optimal antiviral T cell responses during acute LCMV infection.
Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature
◽
10.3410/f.718342706.793494594
◽
2014
◽
Author(s):
Thomas Huenig
Keyword(s):
T Cells
◽
T Cell
◽
Regulatory T Cells
◽
T Cell Responses
◽
Type I Interferons
◽
Type I
◽
Cell Responses
◽
Lcmv Infection
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Ultrasound findings of finger, wrist and knee joints in Mucopolysaccharidosis Type I
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2021.05.009
◽
2021
◽
Author(s):
Johannes Roth
◽
Michal Inbar-Feigenberg
◽
Julian Raiman
◽
Marg Bisch
◽
Pranesh Chakraborty
◽
...
Keyword(s):
Knee Joints
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Ultrasound Findings
Download Full-text
Phase I/II clinical trial design for a novel therapy for mucopolysaccharidosis type I with an intravenously administered blood-brain barrier-crossing enzyme (JR-171)
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2020.12.105
◽
2021
◽
Vol 132
(2)
◽
pp. S49
Author(s):
Ryo Higurashi
◽
Mika Okazaki
◽
Satoshi Kawashima
◽
Toshiaki Ikeda
◽
Sairei So
◽
...
Keyword(s):
Clinical Trial
◽
Phase I
◽
Blood Brain Barrier
◽
Trial Design
◽
Clinical Trial Design
◽
Brain Barrier
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Novel Therapy
Download Full-text
Sexual behaviour in a murine model of mucopolysaccharidosis type I (MPS I)
PLoS ONE
◽
10.1371/journal.pone.0220429
◽
2019
◽
Vol 14
(12)
◽
pp. e0220429
◽
Cited By ~ 1
Author(s):
Ana Barbosa Mendes
◽
Cinthia Castro do Nascimento
◽
Vânia D’Almeida
Keyword(s):
Sexual Behaviour
◽
Murine Model
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Mps I
Download Full-text
430. Antibodies to Recombinant Human α-L-Iduronidase Enhance Uptake into Macrophages in Murine Mucopolysaccharidosis Type I
Molecular Therapy
◽
10.1016/s1525-0016(16)33239-7
◽
2016
◽
Vol 24
◽
pp. S170-S171
Author(s):
Valentina Sanghez
◽
Shih-Hsin Kan
◽
Steven Q Le
◽
Don Clarke
◽
Kristen Vondrak
◽
...
Keyword(s):
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
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Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB
PLoS ONE
◽
10.1371/journal.pone.0035787
◽
2012
◽
Vol 7
(4)
◽
pp. e35787
◽
Cited By ~ 103
Author(s):
Fiona L. Wilkinson
◽
Rebecca J. Holley
◽
Kia J. Langford-Smith
◽
Soumya Badrinath
◽
Aiyin Liao
◽
...
Keyword(s):
Mouse Models
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
Anti-drug antibody response in mucopolysaccharidosis type I patients treated with AGT-181, a brain penetrating human insulin receptor antibody-iduronidase fusion protein
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2017.12.343
◽
2018
◽
Vol 123
(2)
◽
pp. S126
◽
Cited By ~ 3
Author(s):
Mathias Schmidt
◽
Ruben J. Boado
◽
Roberto Giugliani
◽
William M. Pardridge
Keyword(s):
Antibody Response
◽
Fusion Protein
◽
Insulin Receptor
◽
Human Insulin
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Receptor Antibody
◽
Human Insulin Receptor
Download Full-text
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