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Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement

Frontiers in molecular biosciences, 2021-09, Vol.8, p.624988-624988

2021

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An international study of caregiver-reported burden and quality of life in metachromatic leukodystrophy

Orphanet journal of rare diseases, 2022-09, Vol.17 (1), p.1-329, Article 329

2022

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Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study

Orphanet journal of rare diseases, 2023-08, Vol.18 (1), p.238-238, Article 238

2023

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Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Frontiers in molecular neuroscience, 2021-05, Vol.14, p.677895-677895

2021

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Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries

Orphanet journal of rare diseases, 2024-02, Vol.19 (1), p.46-46, Article 46

2024

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Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy

Orphanet journal of rare diseases, 2022-10, Vol.17 (1), p.370-19, Article 370

2022

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Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Orphanet journal of rare diseases, 2022-02, Vol.17 (1), p.48-48, Article 48

2022

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Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study

Orphanet journal of rare diseases, 2023-07, Vol.18 (1), p.204-204, Article 204

2023

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Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.771-784

2020

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

Human gene therapy, 2012-08, Vol.23 (8), p.903-914

2012

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Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

Molecular therapy. Methods & clinical development, 2024-06, Vol.32 (2), p.101248-101248, Article 101248

2024

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Autosomal recessive cerebellar ataxia caused by mutations in the PEX2 gene

Orphanet journal of rare diseases, 2011-03, Vol.6 (1), p.8-8, Article 8

2011

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Hematopoietic stem cell transplantation in leukodystrophies

Handbook of clinical neurology, 2024, Vol.204, p.355

2024

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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

Human gene therapy. Clinical development, 2014-09, Vol.25 (3), p.164-177

2014

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Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy

Human molecular genetics, 2006-01, Vol.15 (1), p.53-64

2006

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Intralysosomal Cystine Accumulation in Mice Lacking Cystinosin, the Protein Defective in Cystinosis

Molecular and Cellular Biology, 2002-11, Vol.22 (21), p.7622-7632

2002

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Gene therapy for neurodegenerative disorders in children: dreams and realities

Archives de pédiatrie : organe officiel de la Société française de pédiatrie, 2023-11, Vol.30 (8), p.8-8S40

2023

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Intravenous gene therapy using AAVPHP.eB for metachromatic leukodystrophy

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107758, Article 107758

2024

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Cell-based device provides effective therapeutic strategy to treat metachromatic leukodystrophy

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107759, Article 107759

2024

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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

The New England journal of medicine, 2017-10, Vol.377 (17), p.1630-1638

2017

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Heterogeneity of persistent hyperinsulinaemic hypoglycaemia. A series of 175 cases

European journal of pediatrics, 2002-10, Vol.161 (1), p.37-48

2002

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Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial

Molecular genetics and metabolism, 2020-09, Vol.131 (1-2), p.235-244

2020

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LC-MS/MS quantification of three C16 sulfatide species in dried blood spots for the diagnosis and treatment monitoring of metachromatic leukodystrophy

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107265, Article 107265

2023

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The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

Human gene therapy, 2021-04, Vol.32 (7-8), p.349-374

2021

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Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement

An international study of caregiver-reported burden and quality of life in metachromatic leukodystrophy

Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study

Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries

Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy

Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study

Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?

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

Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

Autosomal recessive cerebellar ataxia caused by mutations in the PEX2 gene

Hematopoietic stem cell transplantation in leukodystrophies

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

Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy

Intralysosomal Cystine Accumulation in Mice Lacking Cystinosin, the Protein Defective in Cystinosis

Gene therapy for neurodegenerative disorders in children: dreams and realities

Intravenous gene therapy using AAVPHP.eB for metachromatic leukodystrophy

Cell-based device provides effective therapeutic strategy to treat metachromatic leukodystrophy

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Heterogeneity of persistent hyperinsulinaemic hypoglycaemia. A series of 175 cases

Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: Results from a phase 1/2 clinical trial

LC-MS/MS quantification of three C16 sulfatide species in dried blood spots for the diagnosis and treatment monitoring of metachromatic leukodystrophy

International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy: A Consensus-Based Approach

The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

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