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Gene therapy review: Duchenne muscular dystrophy case study

Revue neurologique, 2023-01, Vol.179 (1-2), p.90-105

2023

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GRP78 clustering at the cell surface of neurons transduces the action of exogenous alpha-synuclein

Cell death and differentiation, 2014-12, Vol.21 (12), p.1971-1983

2014

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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

Journal of inherited metabolic disease, 2024-01, Vol.47 (1), p.119-134

2024

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Peripheral immunization of mice to produce antibodies against NMDA receptor as a potential approach to create a rodent model of schizophrenia

European neuropsychopharmacology, 2016-10, Vol.26, p.S271-S272

2016

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Selective disruption of the E-cadherin-catenin system by an algal toxin

British journal of cancer, 2004-03, Vol.90 (5), p.1100-1107

2004

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Exogenous -Synuclein Decreases Raft Partitioning of Cav2.2 Channels Inducing Dopamine Release

The Journal of neuroscience, 2014-08, Vol.34 (32), p.10603-10615

2014

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Combination Therapy Is the New Gene Therapy?

Molecular therapy, 2018-01, Vol.26 (1), p.12-14

2018

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SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson’s Disease

Molecular therapy, 2020-02, Vol.28 (2), p.642-652

2020

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Progress and challenges of gene therapy for Pompe disease

Annals of translational medicine, 2019-07, Vol.7 (13), p.287-287

2019

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Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice

Journal of hepatology, 2019-07, Vol.71 (1), p.153-162

2019

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Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors

Hepatology (Baltimore, Md.), 2017-07, Vol.66 (1), p.252-265

2017

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P.1.h.007 - Peripheral immunization of mice to produce antibodies against NMDA receptor as a potential approach to create a rodent model of schizophrenia

European neuropsychopharmacology, 2016-10, Vol.26, p.S271-S272

2016

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Vagueness: A Guide

Ronzitti, Giuseppina

1. Aufl., 2011

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Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment

Acta neuropathologica communications, 2019-10, Vol.7 (1), p.167-167, Article 167

2019

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Azaspiracid-1 Alters the E-cadherin Pool in Epithelial Cells

Toxicological sciences, 2007-02, Vol.95 (2), p.427-435

2007

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O.6Pre-clinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

Neuromuscular disorders : NMD, 2019-10, Vol.29, p.S39-S39

2019

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241st ENMC international workshop: Towards a European unifying lab for Kennedy's disease. 15–17th February, 2019 Hoofddorp, The Netherlands

Neuromuscular disorders : NMD, 2019-09, Vol.29 (9), p.716-724

2019

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Thérapie génique, une nouvelle stratégie pour l’hypophosphatémie liée à l’X

Annales d'endocrinologie, 2021-10, Vol.82 (5), p.255-256

2021

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TH.O.18 - Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S246-S246

2017

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Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S246

2017

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Yessotoxin induces the accumulation of altered E-cadherin dimers that are not part of adhesive structures in intact cells

Toxicology (Amsterdam), 2008-02, Vol.244 (2), p.145-156

2008

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Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S246

2017

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TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

Neuromuscular disorders : NMD, 2017-10, Vol.27, p.S246-S246

2017

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Protein markers of algal toxin contamination in shellfish

Toxicon (Oxford), 2008-11, Vol.52 (6), p.705-713

2008

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The total activity of a mixture of okadaic acid-group compounds can be calculated by those of individual analogues in a phosphoprotein phosphatase 2A assay

Toxicon (Oxford), 2009-05, Vol.53 (6), p.631-637

2009

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Gene therapy review: Duchenne muscular dystrophy case study

GRP78 clustering at the cell surface of neurons transduces the action of exogenous alpha-synuclein

Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice

Peripheral immunization of mice to produce antibodies against NMDA receptor as a potential approach to create a rodent model of schizophrenia

Selective disruption of the E-cadherin-catenin system by an algal toxin

Exogenous -Synuclein Decreases Raft Partitioning of Cav2.2 Channels Inducing Dopamine Release

Combination Therapy Is the New Gene Therapy?

SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson’s Disease

Progress and challenges of gene therapy for Pompe disease

Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice

Autophagy determines efficiency of liver‐directed gene therapy with adeno‐associated viral vectors

P.1.h.007 - Peripheral immunization of mice to produce antibodies against NMDA receptor as a potential approach to create a rodent model of schizophrenia

Vagueness: A Guide

Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment

Azaspiracid-1 Alters the E-cadherin Pool in Epithelial Cells

O.6Pre-clinical development of SPK-3006, an investigational liver-directed AAV gene therapy for the treatment of Pompe disease

241st ENMC international workshop: Towards a European unifying lab for Kennedy's disease. 15–17th February, 2019 Hoofddorp, The Netherlands

Thérapie génique, une nouvelle stratégie pour l’hypophosphatémie liée à l’X

TH.O.18 - Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease

Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease

Yessotoxin induces the accumulation of altered E-cadherin dimers that are not part of adhesive structures in intact cells

Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes

Protein markers of algal toxin contamination in shellfish

The total activity of a mixture of okadaic acid-group compounds can be calculated by those of individual analogues in a phosphoprotein phosphatase 2A assay

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