(Q34281044)

1 August 2017

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title

Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy (English)

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lysosomal storage disease

1 August 2017

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ORCID Public Data File 2021

author name string

Platt FM

1

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1 August 2017

Jeyakumar M

2

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1 August 2017

Andersson U

3

1 reference

1 August 2017

Priestman DA

4

1 reference

1 August 2017

Dwek RA

5

1 reference

1 August 2017

Butters TD

6

1 reference

1 August 2017

Cox TM

7

1 reference

1 August 2017

Lachmann RH

8

1 reference

1 August 2017

Hollak C

9

1 reference

1 August 2017

Aerts JM

10

1 reference

1 August 2017

Van Weely S

11

1 reference

1 August 2017

Hrebícek M

12

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1 August 2017

Moyses C

13

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1 August 2017

Gow I

14

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1 August 2017

Elstein D

15

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1 August 2017

Zimran A

16

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1 August 2017

1 April 2001

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Journal of Inherited Metabolic Disease

1 August 2017

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1 August 2017

24

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1 August 2017

2

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1 August 2017

275-290

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https://scigraph.springernature.com/pub.10.1023/a:1010335505357

1 August 2017

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cites work

Extensive Glycosphingolipid Depletion in the Liver and Lymphoid Organs of Mice Treated withN-Butyldeoxynojirimycin

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12 December 2020

Gene therapy at a crossroads

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12 December 2020

Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice

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12 December 2020

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

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12 December 2020

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation.

1 reference

12 December 2020

Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

1 reference

12 December 2020

Neuropathology of mice with targeted disruption of Hexa gene, a model of Tay-Sachs disease

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12 December 2020

Clinical and biochemical outcome of marrow transplantation for Gaucher disease of the Norrbottnian type

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12 December 2020

Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases

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12 December 2020

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

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12 December 2020

Animal models of lysosomal disease: an overview

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12 December 2020

Human neural stem cells: isolation, expansion and transplantation

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12 December 2020

Substrate deprivation: a new therapeutic approach for the glycosphingolipid lysosomal storage diseases.

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12 December 2020

Partial enzyme deficiencies: residual activities and the development of neurological disorders

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12 December 2020

N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.

1 reference

12 December 2020

Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin

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12 December 2020

Treatment of Gaucher disease with an enzyme inhibitor

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12 December 2020

Ten years' experience of bone marrow transplantation for Gaucher disease.

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12 December 2020

Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism

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12 December 2020

The safety and efficacy of combination N-butyl-deoxynojirimycin (SC-48334) and zidovudine in patients with HIV-1 infection and 200-500 CD4 cells/mm3.

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12 December 2020

Lysosomal storage diseases

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12 December 2020

Establishment and properties of neural stem cell clones: plasticity in vitro and in vivo

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12 December 2020

N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo

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12 December 2020

Strategies for targeting therapeutic gene delivery

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12 December 2020

Cellular pathology of lysosomal storage disorders.

1 reference

12 December 2020

Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin.

1 reference

12 December 2020

Prevalence of lysosomal storage disorders

1 reference

12 December 2020

Preparation of the active isomer of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol, inhibitor of murine glucocerebroside synthetase

1 reference

12 December 2020

Modulation of cell-surface transferrin receptor by the imino sugar N-butyldeoxynojirimycin.

1 reference

12 December 2020

N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing

1 reference

12 December 2020

Targeted disruption of the Hexa gene results in mice with biochemical and pathologic features of Tay-Sachs disease

1 reference

12 December 2020

Pathologic gene expression in Gaucher disease: up-regulation of cysteine proteinases including osteoclastic cathepsin K.

1 reference

12 December 2020

New therapeutic prospects for the glycosphingolipid lysosomal storage diseases.

1 reference

12 December 2020

Gene transfer technology in therapy: current applications and future goals

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12 December 2020

Imino sugar therapy for type 1 Gaucher disease

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12 December 2020

Accelerated transport and maturation of lysosomal α–galactosidase A in Fabry lymphoblasts by an enzyme inhibitor

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12 December 2020

Biology and potential strategies for the treatment of GM2 gangliosidoses

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12 December 2020

Generation of specific deoxynojirimycin-type inhibitors of the non-lysosomal glucosylceramidase

1 reference

12 December 2020

Plasma and metabolic abnormalities in Gaucher's disease.

1 reference

12 December 2020

Gene therapy death prompts review of adenovirus vector

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12 December 2020

Mouse models of human lysosomal diseases.

1 reference

12 December 2020

Strange brew: T cells in the liver

1 reference

12 December 2020

Neuronopathic forms of Gaucher's disease

1 reference

12 December 2020

Identifiers

DOI

10.1023/A:1010335505357

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Dimensions Publication ID

1 August 2017

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