Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates (Q42225564)

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scientific article published on 10 April 2014

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English	Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates	scientific article published on 10 April 2014

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scholarly article

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Europe PubMed Central

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19 October 2017

Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates (English)

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Europe PubMed Central

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19 October 2017

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Kevin M. Flanigan

12

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author name string

Darren A Murrey

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Bartholomew J Naughton

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19 October 2017

F Jason Duncan

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19 October 2017

Aaron S Meadows

4

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19 October 2017

Tierra A Ware

5

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19 October 2017

Katie J Campbell

6

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William G Bremer

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19 October 2017

Christopher M Walker

8

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19 October 2017

Laurie Goodchild

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19 October 2017

Brad Bolon

10

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19 October 2017

Krista La Perle

11

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19 October 2017

Douglas M McCarty

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19 October 2017

Haiyan Fu

15

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19 October 2017

publication date

10 April 2014

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19 October 2017

Human gene therapy. Clinical development

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19 October 2017

25

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19 October 2017

2

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19 October 2017

72-84

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19 October 2017

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

1 reference

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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

1 reference

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Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice

1 reference

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AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)

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1 reference

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Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

1 reference

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RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

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Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB

1 reference

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Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice

1 reference

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Sanfilippo syndrome type B, a lysosomal storage disease, is also a tauopathy

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Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

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Innate and adaptive immune activation in the brain of MPS IIIB mouse model.

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Gene therapy using adeno-associated virus vectors

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Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

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CNS-directed gene therapy for lysosomal storage diseases

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Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

1 reference

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1 reference

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26 October 2017

Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis III B.

1 reference

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase

1 reference

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The cryptic life style of adeno-associated virus

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1 reference

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1 reference

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1 reference

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1 reference

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1 reference

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17 August 2018

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1 reference

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17 August 2018

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Mucopolysaccharidosis type IIIB: characterisation and expression of wild-type and mutant recombinant alpha-N-acetylglucosaminidase and relationship with sanfilippo phenotype in an attenuated patient

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https://pubmed.ncbi.nlm.nih.gov/24720466

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Sanfilippo type B syndrome (mucopolysaccharidosis III B): allelic heterogeneity corresponds to the wide spectrum of clinical phenotypes

1 reference

https://pubmed.ncbi.nlm.nih.gov/24720466

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1089/HUMC.2013.208

1 reference

Europe PubMed Central

PMC publication ID

19 October 2017

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

19 October 2017

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

19 October 2017

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