Therapies of mucopolysaccharidosis IVA (Morquio A syndrome). (Q34545883)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

title

Therapies of mucopolysaccharidosis IVA (Morquio A syndrome) (English)

1 reference

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23 February 2020

author

Carlos J Alméciga-Díaz

2

1 reference

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23 February 2020

author name string

Shunji Tomatsu

1

1 reference

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23 February 2020

Hector Barbosa

3

1 reference

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23 February 2020

Adriana M Montaño

4

1 reference

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23 February 2020

Luis A Barrera

5

1 reference

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23 February 2020

Tsutomu Shimada

6

1 reference

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23 February 2020

Eriko Yasuda

7

1 reference

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23 February 2020

William G Mackenzie

8

1 reference

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23 February 2020

Robert W Mason

9

1 reference

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23 February 2020

Yasuyuki Suzuki

10

1 reference

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23 February 2020

Kenji E Orii

11

1 reference

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23 February 2020

Tadao Orii

12

1 reference

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23 February 2020

publication date

1 October 2013

1 reference

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Expert opinion on orphan drugs

23 February 2020

published in

1 reference

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23 February 2020

volume

1

1 reference

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23 February 2020

issue

10

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23 February 2020

page(s)

805-818

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Current and emerging treatments and surgical interventions for Morquio A syndrome: a review

23 February 2020

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7 July 2018

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Europe gives gene therapy the green light

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

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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

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Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA.

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SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors

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

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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

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Lentiviral-mediated correction of MPS VI cells and gene transfer to joint tissues.

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Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery

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Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Various cells retrovirally transduced with N-acetylgalactosoamine-6-sulfate sulfatase correct Morquio skin fibroblasts in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enzyme-replacement therapy in life-threatening hypophosphatasia.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

A molecular and histological characterization of cartilage from patients with Morquio syndrome

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Sacral dimple: incidental findings from newborn evaluation. Mucopolysaccharidosis IVA disease

27 October 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Fetal presentation of Morquio disease type A

21 January 2018

1 reference

12 December 2020

Limb-saving medicines sought to prevent amputations

1 reference

12 December 2020

Enzyme replacement therapy for mucopolysaccharidosis II from 3 months of age: a 3-year follow-up

1 reference

12 December 2020

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

1 reference

12 December 2020

[Construction of an adenoassociated, viral derived, expression vector to correct the genetic defect in Morquio A disease]

1 reference

12 December 2020

Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB

1 reference

12 December 2020

10.1517/21678707.2013.846853

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

PubMed publication ID

25419501

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json