Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I. (Q39339697)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28560469%20AND%20SRC:MED&resulttype=core&format=json

21 March 2020

review article

2 references

29 October 2020

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I (English)

1 reference

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21 March 2020

1 reference

29 October 2020

metachromatic leukodystrophy

1 reference

PubMed

lysosomal storage disease

29 October 2020

1 reference

PubMed

29 October 2020

mucopolysaccharidosis I

1 reference

29 October 2020

1

1 reference

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21 March 2020

Francesca Fumagalli

2

1 reference

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21 March 2020

Alessandro Aiuti

5

1 reference

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21 March 2020

author name string

Valeria Calbi

3

1 reference

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21 March 2020

Maria Ester Bernardo

4

1 reference

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21 March 2020

language of work or name

1 reference

30 May 2017

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28560469%20AND%20SRC:MED&resulttype=core&format=json

Journal of Inherited Metabolic Disease

21 March 2020

1 July 2017

1 reference

29 October 2020

2 references

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21 March 2020

29 October 2020

40

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21 March 2020

issue

4

1 reference

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21 March 2020

page(s)

543-554

1 reference

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Creative Commons Attribution 4.0 International

21 March 2020

copyright license

10 references

12 March 2019

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21 March 2019

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on focus list of Wikimedia project

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copyright status

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Innate immune responses in the brain of sphingolipid lysosomal storage diseases

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11 September 2017

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11 September 2017

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11 September 2017

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Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

11 September 2017

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11 September 2017

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Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice

11 September 2017

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11 September 2017

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Storage solutions: treating lysosomal disorders of the brain

11 September 2017

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11 September 2017

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11 September 2017

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11 September 2017

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14 September 2017

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14 September 2017

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14 September 2017

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Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

14 September 2017

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25 September 2017

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Gene therapy for retinal diseases

15 June 2018

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15 June 2018

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Mucopolysaccharidosis VI: pathophysiology, diagnosis and treatment

15 June 2018

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Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience.

21 August 2018

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Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines

21 August 2018

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Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy

21 August 2018

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The natural course of gross motor deterioration in metachromatic leukodystrophy

21 August 2018

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Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy

21 August 2018

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Treatment of infantile Pompe disease with alglucosidase alpha: the UK experience

21 August 2018

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Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

21 August 2018

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21 August 2018

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Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation

21 August 2018

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Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease

21 August 2018

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21 August 2018

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Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

21 August 2018

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Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

21 August 2018

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Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

21 August 2018

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alpha-L-Iduronidase transport in neurites.

21 August 2018

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Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

21 August 2018

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Axons mediate the distribution of arylsulfatase A within the mouse hippocampus upon gene delivery.

21 August 2018

1 reference

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AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.

21 August 2018

1 reference

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Enzyme-replacement therapy in mucopolysaccharidosis I.

21 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5500670

21 August 2018

1 reference

https://pubmed.ncbi.nlm.nih.gov/28560469

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1007/S10545-017-0052-4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28560469%20AND%20SRC:MED&resulttype=core&format=json

21 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28560469%20AND%20SRC:MED&resulttype=core&format=json

21 March 2020

PubMed publication ID

28560469

1 reference