(Q30657387)

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

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Europe PubMed Central

PMC publication ID

10 August 2017

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

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10 August 2017

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Alessandra Ricca

1

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5 June 2020

Francesco Morena

4

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5 June 2020

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5 June 2020

Alessandra Bolino

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5 June 2020

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5 June 2020

Alessandra Biffi

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Alessandra Biffi

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5 June 2020

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Nicole Rufo

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5 June 2020

Silvia Ungari

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5 June 2020

Wilem Kulik

6

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5 June 2020

Valeria Alberizzi

7

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5 June 2020

Francesca Bianchi

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5 June 2020

Ubaldo Del Carro

10

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5 June 2020

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5 March 2015

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10 August 2017

Human Molecular Genetics

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5 June 2020

24

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5 June 2020

12

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5 June 2020

3372-3389

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5 June 2020

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1 reference

Europe PubMed Central

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC4498152/fullTextXML

based on heuristic

inferred from PubMed Central ID database lookup

Extracellular vesicles from neural stem cells transfer IFN-γ via Ifngr1 to activate Stat1 signaling in target cells.

1 reference

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14 July 2018

Early axonal loss accompanied by impaired endocytosis, abnormal axonal transport, and decreased microtubule stability occur in the model of Krabbe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

1 reference

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14 July 2018

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy 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=4498152

14 July 2018

Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Reversibility of neuropathology in Tay-Sachs-related diseases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Bone Marrow Transplantation Alters the Tremor Phenotype in the Murine 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=4498152

14 July 2018

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Region- and age-dependent alterations of glial-neuronal metabolic interactions correlate with CNS pathology 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=4498152

14 July 2018

Multipotent stromal cells alleviate inflammation, neuropathology, and symptoms associated with globoid cell leukodystrophy in the twitcher mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Microglial repopulation model reveals a robust homeostatic process for replacing CNS myeloid cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine 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=4498152

14 July 2018

Activating transcription factor 3 and the nervous system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Psychosine induces the dephosphorylation of neurofilaments by deregulation of PP1 and PP2A phosphatases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Peripheral neuropathy in the Twitcher mouse involves the activation of axonal caspase 3.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Systemic delivery of bone marrow-derived mesenchymal stromal cells diminishes neuropathology in a mouse model of Krabbe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 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=4498152

14 July 2018

Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine 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=4498152

14 July 2018

Gene therapy for leukodystrophies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

A signaling cascade of nuclear calcium-CREB-ATF3 activated by synaptic NMDA receptors defines a gene repression module that protects against extrasynaptic NMDA receptor-induced neuronal cell death and ischemic brain damage.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Mesenchymal lineage stem cells have pronounced anti-inflammatory effects in the twitcher mouse model of Krabbe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Alteration of glial-neuronal metabolic interactions in a mouse model of Alexander disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Infiltrating blood-derived macrophages are vital cells playing an anti-inflammatory role in recovery from spinal cord injury in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Diffusion tensor imaging detects abnormalities in the corticospinal tracts of neonates with infantile Krabbe disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Single-dose intracerebroventricular administration of galactocerebrosidase improves survival 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=4498152

14 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Trends in haematopoietic cell transplantation for inborn errors of metabolism

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Globoid Cell Leukodystrophy (Krabbe's Disease): Update

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

The twitcher mouse: a model for Krabbe disease and for experimental therapies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Progressive accumulation of toxic metabolite in a genetic leukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

The neuropathy of Krabbe's infantile cerebral sclerosis (globoid cell leucodystrophy).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

The twitcher mouse. Central nervous system pathology after bone marrow transplantation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

Lectin histochemistry of glycolipid storage diseases on frozen and paraffin-embedded tissue sections

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4498152

14 July 2018

CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Hematopoietic cell transplantation ameliorates clinical phenotype and progression of the CNS pathology in the mouse model of late onset Krabbe disease

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Biochemical and pathological evaluation of long-lived mice with globoid cell leukodystrophy after bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 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

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

The therapeutic potential of neural stem/progenitor cells in murine globoid cell leukodystrophy is conditioned by macrophage/microglia activation

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

The long-term outcomes of presymptomatic infants transplanted for Krabbe disease: report of the workshop held on July 11 and 12, 2008, Holiday Valley, New York

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Substrate-reduction therapy enhances the benefits of bone marrow transplantation in young mice with globoid cell leukodystrophy

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Central nervous system-directed AAV2/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Axonopathy is a compounding factor in the pathogenesis of Krabbe disease.

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Myelin deterioration in Twitcher mice: motor evoked potentials and magnetic resonance imaging as in vivo monitoring tools

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Monitoring disease evolution and treatment response in lysosomal disorders by the peripheral benzodiazepine receptor ligand PK11195.

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Autonomic denervation of lymphoid organs leads to epigenetic immune atrophy in a mouse model of Krabbe disease.

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Infiltrating monocytes trigger EAE progression, but do not contribute to the resident microglia pool.

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Immunohistological study of globoid cell leukodystrophy

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Activation transcription factor-3 activation and the development of spinal cord degeneration in a rat model of amyotrophic lateral sclerosis

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Effects of developmental age, brain region, and time in culture on long-term proliferation and multipotency of neural stem cell populations.

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Specific determination of beta-galactocerebrosidase activity via competitive inhibition of beta-galactosidase

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

P(0) glycoprotein overexpression causes congenital hypomyelination of peripheral nerves

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV086

21 January 2018

Identification of hematopoietic stem cell-specific miRNAs enables gene therapy 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=4498152

25 November 2018

ATF3 expression precedes death of spinal motoneurons in amyotrophic lateral sclerosis-SOD1 transgenic mice and correlates with c-Jun phosphorylation, CHOP expression, somato-dendritic ubiquitination and Golgi fragmentation

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

L-cycloserine slows the clinical and pathological course in mice with globoid cell leukodystrophy (twitcher mice)

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Expression of immune-related molecules is downregulated in twitcher mice following bone marrow transplantation

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Peripheral neuropathy in Krabbe disease: electrodiagnostic findings

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Peripheral neuropathy in Krabbe disease: effect of hematopoietic stem cell transplantation

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Pathology of the peripheral nerve in the twitcher mouse following bone marrow transplantation

1 reference

https://pubmed.ncbi.nlm.nih.gov/25749991

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1093/HMG/DDV086

1 reference

Europe PubMed Central

PMC publication ID

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25749991%20AND%20SRC:MED&resulttype=core&format=json

5 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:25749991%20AND%20SRC:MED&resulttype=core&format=json

5 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:25749991%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

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