rdf:type |
|
lifeskim:mentions |
umls-concept:C0011209,
umls-concept:C0026703,
umls-concept:C0026809,
umls-concept:C0086022,
umls-concept:C0205171,
umls-concept:C0302350,
umls-concept:C0332307,
umls-concept:C0442335,
umls-concept:C0521026,
umls-concept:C0814225,
umls-concept:C0927232,
umls-concept:C1150069,
umls-concept:C1512924,
umls-concept:C1633983,
umls-concept:C1705099
|
pubmed:issue |
7
|
pubmed:dateCreated |
2010-7-12
|
pubmed:abstractText |
Finding efficient central nervous system (CNS) delivery approaches has been the major challenge facing therapeutic development for treating diseases with global neurological manifestation, such as mucopolysaccharidosis (MPS) IIIB, a lysosomal storage disease, caused by autosomal recessive defect of alpha-N-acetylglucosaminidase (NaGlu). Previously, we developed an approach, intracisternal (i.c.) injection, to deliver recombinant adeno-associated viral (rAAV) vector to the CNS of mice, leading to a widespread periventricular distribution of transduction.
|
pubmed:grant |
|
pubmed:language |
eng
|
pubmed:journal |
|
pubmed:citationSubset |
IM
|
pubmed:chemical |
|
pubmed:status |
MEDLINE
|
pubmed:month |
Jul
|
pubmed:issn |
1521-2254
|
pubmed:author |
|
pubmed:issnType |
Electronic
|
pubmed:volume |
12
|
pubmed:owner |
NLM
|
pubmed:authorsComplete |
Y
|
pubmed:pagination |
624-33
|
pubmed:meshHeading |
pubmed-meshheading:20603889-Acetylglucosaminidase,
pubmed-meshheading:20603889-Animals,
pubmed-meshheading:20603889-Behavior, Animal,
pubmed-meshheading:20603889-Brain,
pubmed-meshheading:20603889-Cerebral Ventricles,
pubmed-meshheading:20603889-Cognition,
pubmed-meshheading:20603889-Dependovirus,
pubmed-meshheading:20603889-Disease Progression,
pubmed-meshheading:20603889-Gene Transfer Techniques,
pubmed-meshheading:20603889-Genetic Vectors,
pubmed-meshheading:20603889-Genome, Viral,
pubmed-meshheading:20603889-Lysosomes,
pubmed-meshheading:20603889-Mice,
pubmed-meshheading:20603889-Mucopolysaccharidosis III,
pubmed-meshheading:20603889-Recombinant Proteins,
pubmed-meshheading:20603889-Recombination, Genetic,
pubmed-meshheading:20603889-Survival Analysis
|
pubmed:year |
2010
|
pubmed:articleTitle |
Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery.
|
pubmed:affiliation |
Center for Gene Therapy, Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.
|
pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't,
Research Support, N.I.H., Extramural
|