Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy. (Q41619077)

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16 February 2020

title

Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy (English)

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16 February 2020

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Ana Del Pozo-Rodriguez

3

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16 February 2020

author name string

Aritz Pérez Ruiz de Garibay

1

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16 February 2020

Diego Delgado

2

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16 February 2020

María Ángeles Solinís

4

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16 February 2020

Alicia Rodríguez Gascón

5

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16 February 2020

language of work or name

English

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publication date

26 October 2012

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Drug Design, Development and Therapy

16 February 2020

published in

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16 February 2020

volume

6

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16 February 2020

page(s)

303-310

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Creative Commons Attribution-NonCommercial 3.0 Unported

16 February 2020

copyright license

start time

1 October 2012

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April 2022 Public Data File from Crossref

copyright status

copyrighted

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cites work

Lysosomal delivery of therapeutic enzymes in cell models of Fabry disease

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Anti-α-galactosidase A antibody response to agalsidase beta treatment: data from the Fabry Registry

6 June 2018

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Dextran-protamine-solid lipid nanoparticles as a non-viral vector for gene therapy: in vitro characterization and in vivo transfection after intravenous administration to mice

6 June 2018

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Update on role of agalsidase alfa in management of Fabry disease

6 June 2018

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Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer.

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Solid lipid nanoparticles as potential tools for gene therapy: in vivo protein expression after intravenous administration.

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A proline-rich peptide improves cell transfection of solid lipid nanoparticle-based non-viral vectors

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Solid lipid nanoparticles for retinal gene therapy: transfection and intracellular trafficking in RPE cells.

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Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg

6 June 2018

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Correction of cardiac abnormalities in fabry mice by direct intraventricular injection of a recombinant lentiviral vector that engineers expression of alpha-galactosidase A.

6 June 2018

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The internalization route resulting in successful gene expression depends on both cell line and polyethylenimine polyplex type

6 June 2018

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Gene therapy for lysosomal storage diseases.

6 June 2018

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The probable structure of the protamine-DNA complex.

6 June 2018

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Cell type-specific gene expression, mediated by TFL-3, a cationic liposomal vector, is controlled by a post-transcription process of delivered plasmid DNA.

6 June 2018

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Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer

6 June 2018

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Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

6 June 2018

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Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer

6 June 2018

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Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

6 June 2018

1 reference

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Purification and properties of the two major isozymes of alpha-galactosidase from human placenta

6 June 2018

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Understanding the mechanism of protamine in solid lipid nanoparticle-based lipofection: The importance of the entry pathway

6 June 2018

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Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period

19 August 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3484727

Evaluation of nuclear transfer and transcription of plasmid DNA condensed with protamine by microinjection: the use of a nuclear transfer score

19 August 2018

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Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3484727

AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.

19 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3484727

Enzymatic Defect in Fabry's Disease

19 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23118528

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Differential assay for lysosomal alpha-galactosidases in human tissues and its application to Fabry's disease

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23118528

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Solid lipid nanoparticles: formulation factors affecting cell transfection capacity

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23118528

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.2147/DDDT.S36131

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23118528 AND SRC:MED&resulttype=core&format=json

16 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23118528 AND SRC:MED&resulttype=core&format=json

16 February 2020

PubMed publication ID

23118528

1 reference