Delivery of lysosomal enzymes for therapeutic use: glucocerebrosidase as an example (Q36640336)

15 August 2017

1 reference

title

Delivery of lysosomal enzymes for therapeutic use: glucocerebrosidase as an example (English)

1 reference

15 August 2017

Gregory A Grabowski

1

1 reference

PubMed ID

17076599

15 August 2017

language of work or name

English

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publication date

1 November 2006

1 reference

Expert Opinion on Drug Delivery

15 August 2017

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15 August 2017

3

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15 August 2017

6

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15 August 2017

771-782

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Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

15 August 2017

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Failure of resting echocardiography and cardiac catheterization to identify pulmonary hypertension in two patients with type I Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

The fine structure of the cerebroside occurring in Gaucher's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Quantitative correlation between the residual activity of beta-hexosaminidase A and arylsulfatase A and the severity of the resulting lysosomal storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

The defect in Hurler and Hunter syndromes. II. Deficiency of specific factors involved in mucopolysaccharide degradation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

The human glucocerebrosidase gene and pseudogene: structure and evolution

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Regulation of the human acid beta-glucosidase promoter in multiple cell types

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Regulation of expression of the gene encoding human acid beta-glucosidase in different cell types

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Translation modulation of acid beta-glucosidase in HepG2 cells: participation of the PKC pathway

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Reconstitution of TCP80/NF90 translation inhibition activity in insect cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Translational inefficiency of acid beta-glucosidase mRNA in transgenic mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Human acid beta-glucosidase: glycosylation is required for catalytic activity

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Identification of the macrophage mannose receptor as a 175-kDa membrane protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Molecular characterization of the human macrophage mannose receptor: demonstration of multiple carbohydrate recognition-like domains and phagocytosis of yeasts in Cos-1 cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Acid beta-glucosidase: enzymology and molecular biology of Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Gaucher's disease: molecular, genetic and enzymological aspects

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Acid beta-glucosidase: intrinsic fluorescence and conformational changes induced by phospholipids and saposin C.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Molecular cloning of a human co-beta-glucosidase cDNA: evidence that four sphingolipid hydrolase activator proteins are encoded by single genes in humans and rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Coding of two sphingolipid activator proteins (SAP-1 and SAP-2) by same genetic locus

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Degradation of membrane-bound ganglioside GM1. Stimulation by bis(monoacylglycero)phosphate and the activator proteins SAP-B and GM2-AP.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

X-ray structure of human acid-β-glucosidase, the defective enzyme in Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

X-ray structure of human acid-beta-glucosidase covalently bound to conduritol-B-epoxide. Implications for Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Analyses of variant acid beta-glucosidases: effects of Gaucher disease mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Molecular cloning and nucleotide sequence of human glucocerebrosidase cDNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Mannose receptor-mediated regulation of serum glycoprotein homeostasis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

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

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Viable mouse models of acid beta-glucosidase deficiency: the defect in Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Immunoelectron microscopic localization of mannose-terminal glucocerebrosidase in lysosomes of rat liver Kupffer cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Turnover and distribution of intravenously administered mannose-terminated human acid beta-glucosidase in murine and human tissues.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

The role of mannosylated enzyme and the mannose receptor in enzyme replacement therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Therapeutic goals in the treatment of Gaucher disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Enzyme therapy of gaucher disease: clinical and biochemical changes during production of and tolerization for neutralizing antibodies.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Management of neutralizing antibody to Ceredase in a patient with type 3 Gaucher disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Beneficial effect of pre-transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Gaucher disease: retrovirus-mediated correction of the enzymatic defect in cultured cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Chemical chaperones--a new concept in drug research

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Chemical chaperones increase the cellular activity of N370S beta -glucosidase: a therapeutic strategy for Gaucher disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Gaucher disease-associated glucocerebrosidases show mutation-dependent chemical chaperoning profiles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

Norrbottnian type of Gaucher disease--clinical, biochemical and molecular biology aspects: successful treatment with bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F17425247.3.6.771

21 January 2018

Identifiers

DOI

10.1517/17425247.3.6.771

1 reference

15 August 2017

1 reference

PubMed ID

17076599