706. Non-Invasive Intranasal Administration of AAV9-Iduronidase Prevents Emergence of Neurologic Disease and Neurocognitive Dysfunction in a Murine Model of Mucopolysaccharidosis Type I

Neurologic Disease ◽

Neurocognitive Dysfunction ◽

Non Invasive

Intranasal gene delivery of AAV9 iduronidase: A non-invasive and effective gene therapy approach for prevention of neurologic disease in a murine model of mucopolysaccharidosis type I

10.1016/j.ymgme.2015.12.195 ◽

2016 ◽

Vol 117 (2) ◽

pp. S26

Author(s):

Lalitha Belur ◽

Megan Buckvold ◽

Kelly M. Podetz-Pedersen ◽

Maureen Riedl ◽

Lucy Vulchanova ◽

...

Keyword(s):

Gene Therapy ◽

Gene Delivery ◽

Murine Model ◽

Type I ◽

Neurologic Disease ◽

Therapy Approach ◽

Gene Therapy Approach ◽

Non Invasive

610. High Level Expression of Human Iduronidase Throughout the Brain in a Murine Model of Mucopolysaccharidosis Type I After Non-Invasive AAV-Mediated Gene Delivery to the CNS

Molecular Therapy ◽

10.1016/s1525-0016(16)35623-4 ◽

2014 ◽

Vol 22 ◽

pp. S236 ◽

Cited By ~ 1

Keyword(s):

Gene Delivery ◽

Murine Model ◽

Type I ◽

High Level Expression ◽

Non Invasive ◽

High Level ◽

The Brain

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 ◽

Chester B. Whitley ◽

...

Keyword(s):

Gene Transfer ◽

Murine Model ◽

Direct Gene Transfer ◽

Type I ◽

Neurologic Disease ◽

Direct Gene

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 ◽

Mps I

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

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 ◽

Adeno Associated Virus

879. Direct Administration of AAV Vectors Encoding Human α-L-Iduronidase to the CNS in a Murine Model of Mucopolysaccharidosis Type I

Molecular Therapy ◽

10.1016/s1525-0016(16)40282-0 ◽

2008 ◽

Vol 16 ◽

pp. S328

Keyword(s):

Murine Model ◽

Type I ◽

Direct Administration

PEGylated cationic nanoemulsions can efficiently bind and transfect pIDUA in a mucopolysaccharidosis type I murine model

Journal of Controlled Release ◽

10.1016/j.jconrel.2015.04.013 ◽

2015 ◽

Vol 209 ◽

pp. 37-46 ◽

Cited By ~ 12

Author(s):

Michelle Fraga ◽

Fernanda Bruxel ◽

Dirnete Diel ◽

Talita Giacomet de Carvalho ◽

Carlos Alberto Perez ◽

...

Keyword(s):

Murine Model ◽

Type I ◽

Mucopolysaccharidosis Type I

88. Lentiviral vector optimization for IDUA expression in the murine model of Hurler syndrome (mucopolysaccharidosis type I)

10.1016/j.ymgme.2007.10.100 ◽

2008 ◽

Vol 93 (2) ◽

pp. 36-37

Author(s):

Paul Score ◽

Arthur T. Johnson ◽

Chester B. Whitley

Keyword(s):

Vector Optimization ◽

Murine Model ◽

Lentiviral Vector ◽

Type I ◽

Hurler Syndrome ◽

Mucopolysaccharidosis Type I

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

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 ◽

Intracerebroventricular Administration ◽

Adeno Associated Virus

Genomic instability in blood cells from murine model of mucopolysaccharidosis type I

Journal of Molecular Histology ◽

10.1007/s10735-011-9361-3 ◽

2011 ◽

Vol 42 (6) ◽

pp. 575-578 ◽

Cited By ~ 5

Author(s):

Juliana Noguti ◽

Vanessa Gonçalves Pereira ◽

Ana Maria Martins ◽

Vânia D’Almeida ◽

Daniel Araki Ribeiro

Keyword(s):

Genomic Instability ◽

Murine Model ◽

Blood Cells ◽

Type I ◽

Mucopolysaccharidosis Type I