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

Mark J Osborn; Ron T McElmurry; Christopher J Lees; Anthony P DeFeo; Zhi-Ying Chen; Mark A Kay; Luigi Naldini; Gordon Freeman; Jakub Tolar; Bruce R Blazar

doi:10.1038/mt.2010.249

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

Molecular Therapy ◽

10.1038/mt.2010.249 ◽

2011 ◽

Vol 19 (3) ◽

pp. 450-460 ◽

Cited By ~ 69

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

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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

Human Gene Therapy ◽

10.1089/hum.2006.17.ft-253 ◽

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

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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

Human Gene Therapy ◽

10.1089/hum.2006.17.1112 ◽

2006 ◽

Vol 17 (11) ◽

pp. 1112-1121 ◽

Cited By ~ 33

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

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69. Gene therapy of mucopolysaccharidosis type I: Intraventricular administration of adeno-associated virus vector transducing the human alpha-l-iduronidase gene in a murine model of the disease

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2007.10.081 ◽

2008 ◽

Vol 93 (2) ◽

pp. 31

Author(s):

R. Scott McIvor ◽

Daniel A. Wolf ◽

Zhenhong Nan ◽

Mayra Quito ◽

Kelly Podetz-Pedersen ◽

...

Keyword(s):

Gene Therapy ◽

Murine Model ◽

Virus Vector ◽

Type I ◽

Intraventricular Administration ◽

Mucopolysaccharidosis Type ◽

Mucopolysaccharidosis Type I ◽

Adeno Associated Virus

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

Proceedings of the National Academy of Sciences ◽

10.1073/pnas.1413645111 ◽

2014 ◽

Vol 111 (41) ◽

pp. 14894-14899 ◽

Cited By ~ 24

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

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

Brain Research Bulletin ◽

10.1016/j.brainresbull.2007.07.018 ◽

2007 ◽

Vol 74 (6) ◽

pp. 429-438 ◽

Cited By ~ 24

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

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Investigation of the bone damage in mucopolysaccharidosis type I Hurler Syndrome: Pathophysiological mechanisms and the impact of ex vivo gene therapy

Bone Reports ◽

10.1016/j.bonr.2020.100597 ◽

2020 ◽

Vol 13 ◽

pp. 100597

Author(s):

Sara Penna ◽

Stefania Crippa ◽

Valentina Capo ◽

Ludovica Santi ◽

Roberto Bosotti ◽

...

Keyword(s):

Gene Therapy ◽

Ex Vivo ◽

Type I ◽

Mucopolysaccharidosis Type ◽

Hurler Syndrome ◽

Mucopolysaccharidosis Type I ◽

Pathophysiological Mechanisms ◽

Bone Damage ◽

Ex Vivo Gene Therapy ◽

The Impact

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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

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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

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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

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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

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