(Q36982214)

English

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

scientific article published on 08 June 2016

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scholarly article

1 reference

Europe PubMed Central

17 August 2017

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery (English)

1 reference

Europe PubMed Central

17 August 2017

author name string

Haiyan Fu

1

1 reference

Europe PubMed Central

17 August 2017

Marcela P Cataldi

2

1 reference

Europe PubMed Central

17 August 2017

Tierra A Ware

3

1 reference

Europe PubMed Central

17 August 2017

Kimberly Zaraspe

4

1 reference

Europe PubMed Central

17 August 2017

Aaron S Meadows

5

1 reference

Europe PubMed Central

17 August 2017

Darren A Murrey

6

1 reference

Europe PubMed Central

17 August 2017

Douglas M McCarty

7

1 reference

Europe PubMed Central

17 August 2017

language of work or name

1 reference

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inferred from title

publication date

8 June 2016

1 reference

Europe PubMed Central

17 August 2017

Molecular Therapy. Methods & Clinical Development

1 reference

Europe PubMed Central

17 August 2017

3

1 reference

Europe PubMed Central

17 August 2017

16036

1 reference

Europe PubMed Central

17 August 2017

Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Ocular manifestations as key features for diagnosing mucopolysaccharidoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Sanfilippo syndrome: a mini-review

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Mucopolysaccharidoses and the eye.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Efficient expression of protein coding genes from the murine U1 small nuclear RNA promoters

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Management of mucopolysaccharidosis type III.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Cloning of the sulphamidase gene and identification of mutations in Sanfilippo A syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Human liver sulphamate sulphohydrolase. Determinations of native protein and subunit Mr values and influence of substrate agylcone structure on catalytic properties

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

29 September 2017

Dimethylmethylene blue-based spectrophotometry of glycosaminoglycans in untreated urine: a rapid screening procedure for mucopolysaccharidoses.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

27 June 2018

Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorder

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs--a proof of principle study.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

The conjoint importance of the hippocampus and anterior thalamic nuclei for allocentric spatial learning: evidence from a disconnection study in the rat.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4898406

4 September 2018

Quantification and characterization of glycosaminoglycans at the nanogram level by a combined azure A-silver staining in agarose gels

1 reference

https://pubmed.ncbi.nlm.nih.gov/27331076

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Enzyme replacement reduces neuropathology in MPS IIIA dogs

1 reference

https://pubmed.ncbi.nlm.nih.gov/27331076

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

A fluorimetric enzyme assay for the diagnosis of Sanfilippo disease type A (MPS IIIA)

1 reference

https://pubmed.ncbi.nlm.nih.gov/27331076

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product

1 reference

https://api.crossref.org/works/10.1038%2FMTM.2016.36

7 January 2021

based on heuristic

inferred from DOI database lookup

Identifiers

10.1038/MTM.2016.36

1 reference

Europe PubMed Central

17 August 2017

1 reference

Europe PubMed Central

17 August 2017

1 reference

Europe PubMed Central

17 August 2017

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