Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy (Q34281044)

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English	Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy	scientific article

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scholarly article

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Europe PubMed Central

PubMed publication ID

11405346

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1 August 2017

review article

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Europe PubMed Central

title

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

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PubMed publication ID

11405346

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1 August 2017

main subject

lysosomal storage disease

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author

Robin Lachmann

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ORCID Public Data File 2021

author name string

Platt FM

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1

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PubMed publication ID

11405346

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Jeyakumar M

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PubMed publication ID

11405346

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Andersson U

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11405346

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Priestman DA

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11405346

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Dwek RA

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11405346

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Butters TD

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11405346

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Cox TM

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11405346

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Lachmann RH

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11405346

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Hollak C

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11405346

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Aerts JM

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Van Weely S

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Hrebícek M

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11405346

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Moyses C

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11405346

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Gow I

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11405346

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Elstein D

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11405346

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Zimran A

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11405346

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1 August 2017

publication date

1 April 2001

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11405346

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1 August 2017

published in

Journal of Inherited Metabolic Disease

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11405346

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1 August 2017

volume

24

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11405346

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issue

2

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11405346

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1 August 2017

page(s)

275-290

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11405346

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cites work

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Gene therapy at a crossroads

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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

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PubMed

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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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https://pubmed.ncbi.nlm.nih.gov/11405346

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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

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12 December 2020

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

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PubMed

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12 December 2020

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

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Animal models of lysosomal disease: an overview

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Human neural stem cells: isolation, expansion and transplantation

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Treatment of Gaucher disease with an enzyme inhibitor

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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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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reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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Lysosomal storage diseases

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Strategies for targeting therapeutic gene delivery

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Cellular pathology of lysosomal storage disorders.

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Prevalence of lysosomal storage disorders

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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inferred from PubMed ID database lookup

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

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stated in

PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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New therapeutic prospects for the glycosphingolipid lysosomal storage diseases.

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene transfer technology in therapy: current applications and future goals

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Imino sugar therapy for type 1 Gaucher disease

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Biology and potential strategies for the treatment of GM2 gangliosidoses

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

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Plasma and metabolic abnormalities in Gaucher's disease.

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene therapy death prompts review of adenovirus vector

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

retrieved

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Mouse models of human lysosomal diseases.

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PubMed

reference URL

https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Strange brew: T cells in the liver

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

based on heuristic

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Neuronopathic forms of Gaucher's disease

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PubMed

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https://pubmed.ncbi.nlm.nih.gov/11405346

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12 December 2020

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Identifiers

DOI

10.1023/A:1010335505357

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Europe PubMed Central

PubMed publication ID

11405346

retrieved

1 August 2017

Dimensions Publication ID

1032325156

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PubMed publication ID

11405346

1 reference

stated in

Europe PubMed Central

PubMed publication ID

11405346

retrieved

1 August 2017

 

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