(Q42145545)

English

Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy

scientific article published on 16 September 2016

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scholarly article

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Europe PubMed Central

PMC publication ID

17 October 2017

Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy (English)

1 reference

Europe PubMed Central

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17 October 2017

0 references

Human Induced Pluripotent Stem Cells

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based on heuristic

inferred from title

neural stem cell

1 reference

based on heuristic

inferred from title

2

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Europe PubMed Central

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11 June 2020

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11 June 2020

Marianna Paulis

8

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11 June 2020

Francesco Morena

10

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11 June 2020

Serena Giannelli

11

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11 June 2020

15

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11 June 2020

17

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11 June 2020

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Vasco Meneghini

1

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11 June 2020

Davide Sala

4

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11 June 2020

Silvia De Cicco

5

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11 June 2020

Marco Luciani

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11 June 2020

Chiara Cavazzin

7

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11 June 2020

Wieslawa Mentzen

9

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11 June 2020

Francesca Sanvito

12

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11 June 2020

Anna Villa

13

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11 June 2020

Alessandro Bulfone

14

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11 June 2020

Sabata Martino

16

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11 June 2020

publication date

16 September 2016

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17 October 2017

Stem Cells Translational Medicine

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11 June 2020

6

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11 June 2020

2

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11 June 2020

352-368

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Europe PubMed Central

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11 June 2020

copyright license

Creative Commons Attribution 4.0 International

3 November 2021

1 reference

April 2022 Public Data File from Crossref

copyright status

0 references

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.

1 reference

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29 May 2018

Adult Mammalian Neural Stem Cells and Neurogenesis: Five Decades Later

1 reference

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29 May 2018

Pluripotent stem cell-derived radial glia-like cells as stable intermediate for efficient generation of human oligodendrocytes

1 reference

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29 May 2018

Arylsulfatase A Overexpressing Human iPSC-derived Neural Cells Reduce CNS Sulfatide Storage in a Mouse Model of Metachromatic Leukodystrophy

1 reference

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29 May 2018

Human neural stem cell transplantation in ALS: initial results from a phase I trial

1 reference

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29 May 2018

Ex vivo gene therapy using patient iPSC-derived NSCs reverses pathology in the brain of a homologous mouse model

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

1 reference

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29 May 2018

Induced neural stem cells achieve long-term survival and functional integration in the adult mouse brain

1 reference

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29 May 2018

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

1 reference

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29 May 2018

Clinical translation of human neural stem cells

1 reference

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29 May 2018

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

1 reference

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29 May 2018

How stem cells speak with host immune cells in inflammatory brain diseases

1 reference

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29 May 2018

Neuro-immune interactions of neural stem cell transplants: from animal disease models to human trials

1 reference

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29 May 2018

Induced pluripotent stem cell-derived neural cells survive and mature in the nonhuman primate brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Human iPSC-derived oligodendrocyte progenitor cells can myelinate and rescue a mouse model of congenital hypomyelination

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Human neural stem cells induce functional myelination in mice with severe dysmyelination

1 reference

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29 May 2018

Cross-talk between neural stem cells and immune cells: the key to better brain repair?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Extracellular membrane vesicles and immune regulation in the brain

1 reference

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29 May 2018

Neural stem cell transplantation in central nervous system disorders: from cell replacement to neuroprotection

1 reference

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29 May 2018

Capture of neuroepithelial-like stem cells from pluripotent stem cells provides a versatile system for in vitro production of human neurons

1 reference

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29 May 2018

Brain regeneration in physiology and pathology: the immune signature driving therapeutic plasticity of neural stem cells

1 reference

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29 May 2018

Transgene excision has no impact on in vivo integration of human iPS derived neural precursors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Genomic instability in induced stem cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome)

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Robust generation of oligodendrocyte progenitors from human neural stem cells and engraftment in experimental demyelination models in mice

1 reference

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29 May 2018

Neural stem cell systems: physiological players or in vitro entities?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Genome modification in human embryonic stem cells

1 reference

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29 May 2018

Delayed post-ischaemic neuroprotection following systemic neural stem cell transplantation involves multiple mechanisms

1 reference

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29 May 2018

Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease

1 reference

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29 May 2018

A rosette-type, self-renewing human ES cell-derived neural stem cell with potential for in vitro instruction and synaptic integration

1 reference

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29 May 2018

Sorting of lysosomal proteins

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29 May 2018

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

1 reference

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29 May 2018

Strategies and new developments in the generation of patient-specific pluripotent stem cells

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29 May 2018

CNS-directed gene therapy for lysosomal storage diseases

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29 May 2018

Reprogramming of human somatic cells to pluripotency with defined factors

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29 May 2018

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

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29 May 2018

Human neural stem cells over-expressing VEGF provide neuroprotection, angiogenesis and functional recovery in mouse stroke model

1 reference

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29 May 2018

Lysosomal storage disorders

1 reference

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29 May 2018

Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters.

1 reference

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29 May 2018

Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

1 reference

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29 May 2018

Direct isolation of human central nervous system stem cells

1 reference

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29 May 2018

Isolation and cloning of multipotential stem cells from the embryonic human CNS and establishment of transplantable human neural stem cell lines by epigenetic stimulation

1 reference

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29 May 2018

Crystal structure of human arylsulfatase A: the aldehyde function and the metal ion at the active site suggest a novel mechanism for sulfate ester hydrolysis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

p.Nitrocatechol Sulfate for Arylsulfatase Assay: Detection of Metachromatic Leukodystrophy Variants

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

29 May 2018

Generation and isolation of oligodendrocyte progenitor cells from human pluripotent stem cells.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

17 August 2018

Origin-dependent neural cell identities in differentiated human iPSCs in vitro and after transplantation into the mouse brain.

1 reference

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17 August 2018

Central nervous system stem cell transplantation for children with neuronal ceroid lipofuscinosis.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5442804

17 August 2018

Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy

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17 August 2018

StemCells, Inc.: Clinical trials of stem cell therapies for CNS disorders. Interview with Martin McGlynn.

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17 August 2018

Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells.

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17 August 2018

Molecular and structural analysis of metachromatic leukodystrophy patients in Indian population.

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17 August 2018

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

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17 August 2018

iPSC lines that do not silence the expression of the ectopic reprogramming factors may display enhanced propensity to genomic instability.

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17 August 2018

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

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17 August 2018

Long-term culture and differentiation of CNS precursors derived from anterior human neural rosettes following exposure to ventralizing factors

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17 August 2018

Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates.

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17 August 2018

Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis

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17 August 2018

Multipotential neural precursors transplanted into the metachromatic leukodystrophy brain fail to generate oligodendrocytes but contribute to limit brain dysfunction

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17 August 2018

Human ES cell-derived neural rosettes reveal a functionally distinct early neural stem cell stage

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17 August 2018

Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease.

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17 August 2018

Intrinsic resistance of neural stem cells to toxic metabolites may make them well suited for cell non-autonomous disorders: evidence from a mouse model of Krabbe leukodystrophy.

1 reference

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17 August 2018

Lysosomal sulfatide storage in the brain of arylsulfatase A-deficient mice: cellular alterations and topographic distribution

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17 August 2018

Genetics of metachromatic leukodystrophy.

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17 August 2018

Erratum: Neural stem cell systems: physiological players or in vitro entities?

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21 January 2018

Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort

1 reference

https://pubmed.ncbi.nlm.nih.gov/28191778

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Glycosylation and phosphorylation of arylsulfatase A

1 reference

https://pubmed.ncbi.nlm.nih.gov/28191778

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Homozygote for mutation c.1204 + 1G > A of the ARSA gene presents with a late-infantile form of metachromatic leukodystrophy and a rare MRI white matter lesion type

1 reference

https://pubmed.ncbi.nlm.nih.gov/28191778

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Neural progenitor cells from human induced pluripotent stem cells generated less autogenous immune response

1 reference

https://pubmed.ncbi.nlm.nih.gov/28191778

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Metachromatic leukodystrophy: molecular genetics and an animal model

1 reference

https://pubmed.ncbi.nlm.nih.gov/28191778

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Gene therapy for lysosomal storage disorders

1 reference

https://pubmed.ncbi.nlm.nih.gov/28191778

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.5966/SCTM.2015-0414

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28191778%20AND%20SRC:MED&resulttype=core&format=json

11 June 2020

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28191778%20AND%20SRC:MED&resulttype=core&format=json

11 June 2020

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28191778%20AND%20SRC:MED&resulttype=core&format=json

11 June 2020

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