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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
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References
Long-term correction of murine phenylketonuria by viral gene transfer: liver versus muscle
Journal of Inherited Metabolic Disease
◽
10.1007/s10545-010-9044-3
◽
2010
◽
Vol 33
(6)
◽
pp. 677-680
◽
Cited By ~ 9
Author(s):
Beat Thöny
Keyword(s):
Gene Transfer
◽
Viral Gene
◽
Viral Gene Transfer
Download Full-text
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
152. Long-term biochemical and neurobehavioral correction in a murine model of mucopolysaccharidosis type I following neonatal intracerebroventricular administration of a therapeutic adeno-associated virus vector
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2009.10.169
◽
2010
◽
Vol 99
(2)
◽
pp. S40
Author(s):
Daniel Wolf
◽
Zhenhong Nan
◽
Lalitha Belur
◽
Debra Swanson
◽
Andrew Lenander
◽
...
Keyword(s):
Murine Model
◽
Virus Vector
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Intracerebroventricular Administration
◽
Adeno Associated Virus
Download Full-text
Bull's eye maculopathy and subfoveal deposition in two mucopolysaccharidosis type I patients on long-term enzyme replacement therapy
American Journal of Ophthalmology Case Reports
◽
10.1016/j.ajoc.2017.10.006
◽
2018
◽
Vol 9
◽
pp. 1-6
◽
Cited By ~ 3
Author(s):
Heather G. Mack
◽
R.C. Andrew Symons
◽
Gerard de Jong
Keyword(s):
Enzyme Replacement Therapy
◽
Replacement Therapy
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Enzyme Replacement
◽
Bull’S Eye Maculopathy
◽
Bull's Eye
Download Full-text
Long-term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotype
Journal of Inherited Metabolic Disease
◽
10.1007/bf00711520
◽
1993
◽
Vol 16
(6)
◽
pp. 1024-1033
◽
Cited By ~ 49
Author(s):
J. J. Hopwood
◽
A. Vellodi
◽
H. S. Scott
◽
C. P. Morris
◽
T. Litjens
◽
...
Keyword(s):
Bone Marrow
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
An exploratory study of brain function and structure in mucopolysaccharidosis type I: Long term observations following hematopoietic cell transplantation (HCT)
Molecular Genetics and Metabolism
◽
10.1016/j.ymgme.2012.07.016
◽
2012
◽
Vol 107
(1-2)
◽
pp. 116-121
◽
Cited By ~ 25
Author(s):
Elsa Shapiro
◽
O. Evren Guler
◽
Kyle Rudser
◽
Kathleen Delaney
◽
Kendra Bjoraker
◽
...
Keyword(s):
Cell Transplantation
◽
Brain Function
◽
Exploratory Study
◽
Hematopoietic Cell Transplantation
◽
Hematopoietic Cell
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
Download Full-text
Non-viral gene transfer using sleeping beauty transposition for long-term gene expression in the CNS of neonatal mice
Experimental Neurology
◽
10.1016/j.expneurol.2006.02.035
◽
2006
◽
Vol 198
(2)
◽
pp. 565-566
Author(s):
Z.L. Demorest
◽
J. Ohlfest
◽
W.C. Low
◽
A. Freese
Keyword(s):
Gene Expression
◽
Gene Transfer
◽
Sleeping Beauty
◽
Viral Gene
◽
Neonatal Mice
◽
Viral Gene Transfer
Download Full-text
Long-term memory for aversive training is impaired in Idua−/− mice, a genetic model of mucopolysaccharidosis type I
Brain Research
◽
10.1016/j.brainres.2006.01.008
◽
2006
◽
Vol 1076
(1)
◽
pp. 225-230
◽
Cited By ~ 22
Author(s):
Gustavo Kellermann Reolon
◽
Luisa Macedo Braga
◽
Melissa Camassola
◽
Tatiana Luft
◽
João Antônio Pêgas Henriques
◽
...
Keyword(s):
Genetic Model
◽
Long Term Memory
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Term Memory
Download Full-text
Long-term evolution of mucopolysaccharidosis type I in twins treated with enzyme replacement therapy plus hematopoietic stem cells transplantation
Heliyon
◽
10.1016/j.heliyon.2021.e07740
◽
2021
◽
pp. e07740
Author(s):
Luis M. Carbajal-Rodríguez
◽
Martín Pérez-García
◽
Raymundo Rodríguez-Herrera
◽
Haydeé Salazar Rosales
◽
Alberto Olaya-Vargas
Keyword(s):
Stem Cells
◽
Type I
◽
Mucopolysaccharidosis Type
◽
Mucopolysaccharidosis Type I
◽
Hematopoietic Stem
◽
Enzyme Replacement
◽
Term Evolution
◽
Stem Cells Transplantation
◽
Hematopoietic Stem Cells Transplantation
Download Full-text
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
Download Full-text
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