Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial. (Q40120885)

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25 March 2020

title

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial (English)

1 reference

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mucopolysaccharidosis type IIIB

25 March 2020

main subject

phase I clinical trial

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gene therapy

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phase II clinical trial

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1 reference

4

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25 March 2020

Kumaran Deiva

17

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25 March 2020

author name string

Marc Tardieu

1

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25 March 2020

Michel Zérah

2

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25 March 2020

Marie-Lise Gougeon

3

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25 March 2020

Stéphanie de Bournonville

5

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25 March 2020

Béatrice Husson

6

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25 March 2020

Dimitrios Zafeiriou

7

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25 March 2020

Giancarlo Parenti

8

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25 March 2020

Philippe Bourget

9

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25 March 2020

Béatrice Poirier

10

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25 March 2020

Valérie Furlan

11

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25 March 2020

Cécile Artaud

12

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25 March 2020

Thomas Baugnon

13

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25 March 2020

Thomas Roujeau

14

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25 March 2020

Ronald G Crystal

15

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25 March 2020

Christian Meyer

16

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25 March 2020

Jean-Michel Heard

18

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25 March 2020

language of work or name

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14 July 2017

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25 March 2020

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25 March 2020

volume

16

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25 March 2020

page(s)

712-720

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25 March 2020

issue

9

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Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece

25 March 2020

cites work

1 reference

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7 January 2021

Mucopolysaccharidosis type IIIB may predominantly present with an attenuated clinical phenotype

1 reference

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7 January 2021

Sanfilippo type B syndrome (mucopolysaccharidosis III B): allelic heterogeneity corresponds to the wide spectrum of clinical phenotypes

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7 January 2021

Molecular defects in Sanfilippo syndrome type B (mucopolysaccharidosis IIIB).

1 reference

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7 January 2021

Molecular characterization of 355 mucopolysaccharidosis patients reveals 104 novel mutations

1 reference

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7 January 2021

The molecular basis of Sanfilippo syndrome type B

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7 January 2021

Overview of the mucopolysaccharidoses

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7 January 2021

Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats.

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7 January 2021

Gene therapy for the neurological manifestations in lysosomal storage disorders

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7 January 2021

Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum.

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7 January 2021

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

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7 January 2021

Gene therapy for aromatic L-amino acid decarboxylase deficiency

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7 January 2021

Engineering adeno-associated viruses for clinical gene therapy

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7 January 2021

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

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7 January 2021

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

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7 January 2021

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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7 January 2021

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

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7 January 2021

A model of mucopolysaccharidosis IIIB (Sanfilippo syndrome type IIIB): N‐acetyl‐α‐D‐glucosaminidase deficiency in Schipperke dogs

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7 January 2021

Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis

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7 January 2021

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.

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7 January 2021

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

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7 January 2021

The Neurobehavioral Phenotype in Mucopolysaccharidosis Type IIIB: an Exploratory Study

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7 January 2021

A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.

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7 January 2021

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA.

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7 January 2021

A Multiplex Assay for the Diagnosis of Mucopolysaccharidoses and Mucolipidoses

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7 January 2021

Suppression by thimerosal of ex-vivo CD4+ T cell response to influenza vaccine and induction of apoptosis in primary memory T cells.

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7 January 2021

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7 January 2021

A mild form of Mucopolysaccharidosis IIIB diagnosed with targeted next-generation sequencing of linked genomic regions

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7 January 2021

Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications

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7 January 2021

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Identifiers

DOI

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25 March 2020

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