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373. Expression of Human Iduronidase from Sleeping Beauty Engineered Human B Lymphocytes as a Cellular Therapy for Mucopolysaccharidosis Type I
Molecular Therapy
◽
10.1016/s1525-0016(16)33982-x
◽
2015
◽
Vol 23
◽
pp. S148
Author(s):
Eric J. Herbig
◽
Kendra Hyland
◽
Mei Xu
◽
Rian de Laat
◽
Erik Olson
◽
...
Keyword(s):
B Lymphocytes
◽
Cellular Therapy
◽
Sleeping Beauty
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
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References
Sleeping Beauty engineered human B lymphocytes express therapeutic levels of human iduronidase: A new approach for mucopolysaccharidosis type I
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2014.12.110
◽
2015
◽
Vol 114
(2)
◽
pp. S53-S54
Author(s):
Eric J. Herbig
◽
Mei Xu
◽
Kendra A. Hyland
◽
Erik Olson
◽
Elena Aronovich
◽
...
Keyword(s):
B Lymphocytes
◽
Sleeping Beauty
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
New Approach
Download Full-text
Iduronidase-transposed human B lymphocytes correct enzyme deficiency and glycosaminoglycan storage disease in immunodeficient mucopolysaccharidosis type I mice
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2019.11.276
◽
2020
◽
Vol 129
(2)
◽
pp. S108
Author(s):
R. Scott McIvor
◽
RIan de Laat
◽
Xu Mei
◽
Kole D. Meeker
◽
Monika Swietlicka
◽
...
Keyword(s):
B Lymphocytes
◽
Storage Disease
◽
Enzyme Deficiency
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
745. Gene Therapy of Murine Mucopolysaccharidosis Type I Using the Sleeping Beauty Transposon System
Molecular Therapy
◽
10.1016/s1525-0016(16)39103-1
◽
2009
◽
Vol 17
◽
pp. S284
Keyword(s):
Gene Therapy
◽
Sleeping Beauty
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Sleeping Beauty Transposon
Download Full-text
73 Sleeping Beauty transposon-mediated correction of mucopolysaccharidosis type I
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2007.08.078
◽
2007
◽
Vol 92
(4)
◽
pp. 27
Author(s):
Elena Aronovich
◽
Jason B. Bell
◽
Lalitha R. Belur
Keyword(s):
Sleeping Beauty
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Sleeping Beauty Transposon
Download Full-text
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
Download Full-text
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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