Limited Transgene Immune Response and Long-Term Expression of Humanα-L-Iduronidase in Young Adult Mice with Mucopolysaccharidosis Type I by Liver-Directed Gene Therapy

2006 ◽  
Vol 17 (11) ◽  
pp. 1112-1121 ◽  
Author(s):  
C. Di Domenico ◽  
D. Di Napoli ◽  
E. Gonzalez Y Reyero ◽  
A. Lombardo ◽  
L. Naldini ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Immune Response ◽  
Young Adult ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Adult Mice

Limited Transgene Immune Response and Long-Term Expression of Human ?-L-Iduronidase in Young Adult Mice with Mucopolysaccharidosis Type I by Liver-Directed Gene Therapy

2006 ◽  
Vol 0 (0) ◽  
pp. 061017065132001
Author(s):  
C. Di Domenico ◽  
D. Di Napoli ◽  
E. Gonzalez Y Reyero ◽  
A. Lombardo ◽  
L. Naldini ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Immune Response ◽  
Young Adult ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Adult Mice

scholarly journals Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α-L-Iduronidase in Mice With Mucopolysaccharidosis Type I

2011 ◽  
Vol 19 (3) ◽  
pp. 450-460 ◽  
Author(s):  
Mark J Osborn ◽  
Ron T McElmurry ◽  
Christopher J Lees ◽  
Anthony P DeFeo ◽  
Zhi-Ying Chen ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Immune Modulation ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Minicircle Dna

scholarly journals Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I

2014 ◽  
Vol 111 (41) ◽  
pp. 14894-14899 ◽  
Author(s):  
C. Hinderer ◽  
P. Bell ◽  
B. L. Gurda ◽  
Q. Wang ◽  
J.-P. Louboutin ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I

scholarly journals Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy

2007 ◽  
Vol 74 (6) ◽  
pp. 429-438 ◽  
Author(s):  
Mayra F. Garcia-Rivera ◽  
Leah E. Colvin-Wanshura ◽  
Matthew S. Nelson ◽  
Zhenhong Nan ◽  
Shaukat A. Khan ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Type I ◽  
Mucopolysaccharidosis Type ◽  
Mucopolysaccharidosis Type I ◽  
Human Stem Cell ◽  
Preclinical Testing ◽  
Cell And Gene Therapy

Long-term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotype

1993 ◽  
Vol 16 (6) ◽  
pp. 1024-1033 ◽  
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
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