Gene therapy progress and prospects: gene therapy of lysosomal storage disorders (Q35186114)

7 August 2017

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title

Gene therapy progress and prospects: gene therapy of lysosomal storage disorders (English)

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7 August 2017

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Cheng SH

1

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PubMed ID

12883523

7 August 2017

Smith AE

series ordinal

2

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7 August 2017

1 August 2003

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7 August 2017

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7 August 2017

10

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7 August 2017

16

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7 August 2017

1275-1281

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https://scigraph.springernature.com/pub.10.1038/sj.gt.3302092

7 August 2017

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cites work

Advances in the treatment of lysosomal storage disease

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Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry

21 January 2018

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Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

21 January 2018

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Enzyme replacement therapy in Fabry disease: a randomized controlled trial

21 January 2018

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Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease

21 January 2018

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Preselective gene therapy for Fabry disease

21 January 2018

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Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells

21 January 2018

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Gene therapy using hematopoietic stem cells: Sisyphus approaches the crest

21 January 2018

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Highly efficient gene transfer into baboon marrow repopulating cells using GALV-pseudotype oncoretroviral vectors produced by human packaging cells

21 January 2018

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

21 January 2018

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Demonstration of feasibility of in vivo gene therapy for Gaucher disease using a chemically induced mouse model

21 January 2018

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Lysosomal acid lipase deficiency: correction of lipid storage by adenovirus-mediated gene transfer in mice

21 January 2018

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Gene therapy progress and prospects: nonviral vectors.

21 January 2018

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Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells

21 January 2018

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Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors

21 January 2018

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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

21 January 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

21 January 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

21 January 2018

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Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

21 January 2018

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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

21 January 2018

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

21 January 2018

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

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In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.

21 January 2018

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Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

21 January 2018

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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

21 January 2018

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Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

21 January 2018

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Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy

21 January 2018

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Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy

21 January 2018

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The lung as a route for systemic delivery of therapeutic proteins and peptides

21 January 2018

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Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

21 January 2018

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Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo

21 January 2018

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Adenovirus-transduced lung as a portal for delivering alpha-galactosidase A into systemic circulation for Fabry disease

21 January 2018

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Noninvasive gene transfer to the lung for systemic delivery of therapeutic proteins.

21 January 2018

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Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

21 January 2018

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Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

21 January 2018

1 reference

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In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

Regulated gene expression systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

Gene therapy for lysosomal storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

inferred from DOI database lookup

7 January 2021

based on heuristic

Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

inferred from DOI database lookup

7 January 2021

based on heuristic

Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302092

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/SJ.GT.3302092

1 reference

7 August 2017

1 reference

PubMed ID

12883523