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Intrathecal and intranasal infusion of adeno-associated virus vector: non-invasive routes of administration achieving corrective levels of iduronidase expression throughout the brain for gene therapy of mucopolysaccharidosis type I

Intrathecal and intranasal infusion of adeno-associated virus vector: non-invasive routes of administration achieving corrective levels of iduronidase expression throughout the brain for gene therapy of mucopolysaccharidosis type I

Authors :
Karen Kozarsky
Leah R. Hanson
Daniel A. Wolf
R. Scott McIvor
Kelley F. Kitto
Zhenhong Nan
Lalitha R. Belur
Kelly M. Podetz-Pedersen
William H. Frey
Walter C. Low
Carolyn A. Fairbanks
Source :
Molecular Genetics and Metabolism. 111:S75-S76
Publication Year :
2014
Publisher :
Elsevier BV, 2014.

Details

ISSN :
10967192
Volume :
111
Database :
OpenAIRE
Journal :
Molecular Genetics and Metabolism
Accession number :
edsair.doi...........d14b2c535094e3888486e051e0122f17
Full Text :
https://doi.org/10.1016/j.ymgme.2013.12.174