Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates (Q45863181)

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scientific article published on 11 March 2014

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English	Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates	scientific article published on 11 March 2014

Statements

scholarly article

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates (English)

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

1 reference

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author name string

Darren A Murrey

1

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Bartholomew J Naughton

2

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

F Jason Duncan

3

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Aaron S Meadows

4

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Tierra Amber Ware

5

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Katie Campbell

6

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

William G Bremer

7

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Christopher Walker

8

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Laurie Goodchild

9

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Brad Bolon

10

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Krista La Perle

11

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Kevin Flanigan

12

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Kim L McBride

13

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Douglas M McCarty

14

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Haiyan Fu

15

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17 December 2017

http://europepmc.org/abstract/MED/24617499

publication date

11 March 2014

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Human gene therapy. Clinical development

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

1 reference

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

12 December 2020

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Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

1 reference

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

12 December 2020

based on heuristic

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

1 reference

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

12 December 2020

based on heuristic

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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

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

12 December 2020

based on heuristic

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Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

1 reference

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

12 December 2020

based on heuristic

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AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival

1 reference

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

12 December 2020

based on heuristic

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Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

1 reference

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

12 December 2020

based on heuristic

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Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Attenuated plasticity in neurons and astrocytes in the mouse model of Sanfilippo syndrome type B.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Drug and gene delivery to the brain: the vascular route

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Prevention of neuropathology in the mouse model of Hurler syndrome

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The cryptic life style of adeno-associated virus

1 reference

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

Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Immune responses to AAV in a phase I study for Canavan disease.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

CNS-directed gene therapy for lysosomal storage diseases

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene therapy using adeno-associated virus vectors

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Mucopolysaccharidosis IIIB: oxidative damage and cytotoxic cell involvement in the neuronal pathogenesis.

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

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

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1089/HUM.2013.208

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/24617499

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