(Q37453594)

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Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy

scientific article published on 29 March 2016

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scholarly article

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy (English)

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

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17

object named as

Luigi Naldini

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8

object named as

John Bringas

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Andrea Calabria

object named as

Andrea Calabria

7

0 references

Eugenio Montini

object named as

Eugenio Montini

13

0 references

object named as

Angela Gritti

18

0 references

Claudio Doglioni

object named as

Claudio Doglioni

12

0 references

author name string

Vasco Meneghini

1

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Annalisa Lattanzi

2

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Luigi Tiradani

3

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Gabriele Bravo

4

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Francesco Morena

5

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Francesca Sanvito

6

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

John Bringas

8

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Jeanne M Fisher-Perkins

9

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Jason P Dufour

10

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Kate C Baker

11

1 reference

Europe PubMed Central

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20 August 2017

Bruce A Bunnell

14

1 reference

Europe PubMed Central

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20 August 2017

Krystof Bankiewicz

15

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Europe PubMed Central

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20 August 2017

Sabata Martino

16

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Europe PubMed Central

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20 August 2017

publication date

29 March 2016

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Europe PubMed Central

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20 August 2017

EMBO Molecular Medicine

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Europe PubMed Central

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20 August 2017

8

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

5

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

489-510

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a 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=5128736

23 August 2017

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=5128736

23 August 2017

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

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=5128736

23 August 2017

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=5128736

23 August 2017

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Lysosomal leukodystrophies: Krabbe disease and metachromatic leukodystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

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=5128736

23 August 2017

Long-term follow-up after gene therapy for canavan disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Safety study of adeno-associated virus serotype 2-mediated human acid sphingomyelinase expression 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=5128736

23 August 2017

Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Whole transcriptome characterization of aberrant splicing events induced by lentiviral vector integrations.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Adeno-associated virus vectorology, manufacturing, and clinical applications

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

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=5128736

23 August 2017

Lentiviral vector integration profiles differ in rodent postmitotic tissues

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Engineered lentiviral vector targeting astrocytes in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

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=5128736

23 August 2017

Clinical and immunopathologic alterations in rhesus macaques affected with globoid cell leukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applications

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

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=5128736

23 August 2017

Lysosomal storage disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Convection-enhanced delivery to the central nervous system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

rAAV human trial experience

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

23 August 2017

Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

14 September 2017

Expression and purification of a human, soluble Arylsulfatase A for Metachromatic Leukodystrophy enzyme replacement therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

14 September 2017

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=5128736

14 September 2017

Assessment of the Infant at Risk

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

14 September 2017

Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

28 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

28 September 2017

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

26 June 2018

VISPA: a computational pipeline for the identification and analysis of genomic vector integration sites

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

26 June 2018

Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

26 June 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

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

26 June 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=5128736

26 June 2018

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

26 June 2018

Genetic galactocerebrosidase deficiency (globoid cell leukodystrophy, Krabbe disease) in rhesus monkeys (Macaca mulatta).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

26 June 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=5128736

26 June 2018

A new analytical bench assay for the determination of arylsulfatase a activity toward galactosyl-3-sulfate ceramide: implication for metachromatic leukodystrophy diagnosis.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 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://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Human alpha-iduronidase gene transfer mediated by adeno-associated virus types 1, 2, and 5 in the brain of nonhuman primates: vector diffusion and biodistribution

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Fatty acid formula supplementation and neuromotor development in rhesus monkey neonates

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5128736

2 September 2018

Serial electrophysiologic studies in rhesus monkeys with Krabbe disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/27025653

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Characterization of the rhesus monkey galactocerebrosidase (GALC) cDNA and gene and identification of the mutation causing globoid cell leukodystrophy (Krabbe disease) in this primate

1 reference

https://pubmed.ncbi.nlm.nih.gov/27025653

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Temperament differences between captive Indian and Chinese-Indian hybrid rhesus macaque neonates

1 reference

https://pubmed.ncbi.nlm.nih.gov/27025653

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Murine, canine and non-human primate models of Krabbe disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/27025653

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/27025653

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Behavioral and physiological characteristics of Indian and Chinese-Indian hybrid rhesus macaque infants

1 reference

https://pubmed.ncbi.nlm.nih.gov/27025653

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/27025653

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.15252/EMMM.201505850

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

20 August 2017

ResearchGate publication ID

0 references

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