Absence of metabolic cross-correction in Tay-Sachs cells: implications for gene therapy (Q42817051)

7 November 2017

Absence of metabolic cross-correction in Tay-Sachs cells: implications for gene therapy (English)

1 reference

7 November 2017

0 references

2

1 reference

7 November 2017

Brunella Tancini

3

0 references

Sabata Martino

object named as

Sabata Martino

1

0 references

Sandro Sonnino

object named as

Sandro Sonnino

7

0 references

Claudio Bordignon

6

object named as

Claudio Bordignon

1 reference

7 November 2017

8

Aldo Orlacchio

1 reference

7 November 2017

Giovanni Maria Severini

4

1 reference

PubMed ID

11923278

7 November 2017

Vanna Chigorno

5

1 reference

PubMed ID

11923278

7 November 2017

language of work or name

English

0 references

publication date

28 March 2002

1 reference

Journal of Biological Chemistry

7 November 2017

1 reference

7 November 2017

277

1 reference

7 November 2017

23

1 reference

7 November 2017

20177-20184

1 reference

Biochemical consequences of mutations causing the GM2 gangliosidoses

7 November 2017

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Lysosomal storage diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Particular forms of beta-N-acetylhexosaminidase in human leukaemic cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Evidence for the regulation of beta-N-acetylhexosaminidase expression during pregnancy in the rat.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Direct determination of the substrate specificity of the alpha-active site in heterodimeric beta-hexosaminidase A

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

An enzyme with properties similar to those of beta-N-acetylhexosaminidase S is expressed in the promyelocytic cell line HL-60.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Treatment of HL-60 cells with dimethyl sulphoxide inhibits the formation of beta-N-acetylhexosaminidase S

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Physiological substrates for human lysosomal beta -hexosaminidase S.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Recent advances in the biochemistry of glycosphingolipid metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Gene transfer approaches to the lysosomal storage disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Gene therapy/cell therapy for lysosomal storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

The molecular basis of lysosomal storage diseases and their treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

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/10.1074%2FJBC.M106164200

Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

1H-NMR study on ganglioside amide protons: evidence that the deuterium exchange kinetics are affected by the preparation of samples.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Preparation of radiolabeled gangliosides

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Lysosomal enzyme precursors in human fibroblasts. Activation of cathepsin D precursor in vitro and activity of beta-hexosaminidase A precursor towards ganglioside GM2.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Intermediate forms of human beta-N-acetylhexosaminidase lack activity towards 4-methylumbelliferyl beta-N-acetylglucosaminide 6-sulphate

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Function of the two mannose 6-phosphate receptors in lysosomal enzyme transport

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Retrovirus-mediated enzymatic correction of Tay-Sachs defect in transduced and non-transduced cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Human beta-hexosaminidase alpha chain: coding sequence and homology with the beta chain

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Broad screening test for sphingolipid-storage diseases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Membrane traffic in sphingolipid storage diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Abnormal transport along the lysosomal pathway in mucolipidosis, type IV disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Accumulation of sialic acid in endocytic compartments interferes with the formation of mature lysosomes. Impaired proteolytic processing of cathepsin B in fibroblasts of patients with lysosomal sialic acid storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Adenoviral gene therapy of the Tay-Sachs disease in hexosaminidase A-deficient knock-out mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Correction of acid beta-galactosidase deficiency in GM1 gangliosidosis human fibroblasts by retrovirus vector-mediated gene transfer: higher efficiency of release and cross-correction by the murine enzyme

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Clinical features of and recent advances in therapy for Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Association of gangliosides to fibroblasts in culture: A study performed with GM1 [14C]-labelled at the sialic acid acetyl group

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

β N-acetylhexosaminidases A and S have similar sub-cellular distributions in HL-60 cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Preparation of radioactive gangliosides, 3H or 14C isotopically labeled at oligosaccharide or ceramide moieties

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Distribution of active α- and β-subunits of β-N-acetylhexosaminidase as a function of leukaemic cell types

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

Protein transport from the secretory to the endocytic pathway in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1074%2FJBC.M106164200

21 January 2018

Identifiers

DOI

10.1074/JBC.M106164200

1 reference

7 November 2017

1 reference

PubMed ID

11923278