Gene therapy for metachromatic leukodystrophy

Viral vectors for therapy of neurologic diseases

(2017) Sourav R. Choudhury et al.   NEUROPHARMACOLOGY

Gene therapy for leukodystrophy: progress, challenges and opportunities

(2016) Patrick Aubourg   Expert Opinion on Orphan Drugs

Mutation Update ofARSAandPSAPGenes Causing Metachromatic Leukodystrophy

(2015) Martina Cesani et al.   HUMAN MUTATION

Outcome of Early Juvenile Onset Metachromatic Leukodystrophy After Unrelated Cord Blood Transplantation

(2015) Xuqin Chen et al.   JOURNAL OF CHILD NEUROLOGY

Expanded newborn screening by mass spectrometry: New tests, future perspectives

(2015) Daniela Ombrone et al.   MASS SPECTROMETRY REVIEWS

Disease specific therapies in leukodystrophies and leukoencephalopathies

(2015) Guy Helman et al.   MOLECULAR GENETICS AND METABOLISM

A clinical approach to the diagnosis of patients with leukodystrophies and genetic leukoencephelopathies

(2015) Sumit Parikh et al.   MOLECULAR GENETICS AND METABOLISM

Case definition and classification of leukodystrophies and leukoencephalopathies

(2015) Adeline Vanderver et al.   MOLECULAR GENETICS AND METABOLISM

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

(2015) Michel Zerah et al.   Human Gene Therapy Clinical Development

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

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

The Leukodystrophies

(2014) Hannah Gordon et al.   SEMINARS IN NEUROLOGY

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

(2014) Jonathan B. Rosenberg et al.   Human Gene Therapy Clinical Development

Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy

(2013) Holly R. Martin et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Blood–brain barrier structure and function and the challenges for CNS drug delivery

(2013) N. Joan Abbott   JOURNAL OF INHERITED METABOLIC DISEASE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy

(2013) Martje E. van Egmond et al.   JAMA Neurology

Humoral Immune Response to AAV

(2013) Roberto Calcedo et al.   Frontiers in Immunology

Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice

(2012) Françoise Piguet et al.   HUMAN GENE THERAPY

Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age

(2012) F. Matthes et al.   HUMAN MOLECULAR GENETICS

Immunologic Privilege in the Central Nervous System and the Blood–Brain Barrier

(2012) Leslie L Muldoon et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

Role of sulfatide in normal and pathological cells and tissues

(2012) Tadanobu Takahashi et al.   JOURNAL OF LIPID RESEARCH

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

(2012) Mohammad A Rafi et al.   MOLECULAR THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Gallbladder polyposis in metachromatic leukodystrophy

(2012) Aanchal Agarwal et al.   PEDIATRIC RADIOLOGY

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

(2012) Dolan Sondhi et al.   Human Gene Therapy Methods

Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy

(2011) S. Stroobants et al.   HUMAN MOLECULAR GENETICS

Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course

(2011) Samuel Groeschel et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Developing treatment options for metachromatic leukodystrophy

(2011) Spyros P. Batzios et al.   MOLECULAR GENETICS AND METABOLISM

Glial-derived neurotrophic factor gene transfer for Parkinson's disease: Anterograde distribution of AAV2 vectors in the primate brain

(2011) Adrian P. Kells et al.   NEUROBIOLOGY OF DISEASE

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

(2010) L D de Hosson et al.   BONE MARROW TRANSPLANTATION

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations

(2010) Mark M. Souweidane et al.   Journal of Neurosurgery-Pediatrics

Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: A 5-year follow-up in three affected siblings

(2010) Casey Cable et al.   MOLECULAR GENETICS AND METABOLISM

Metachromatic Leukodystrophy – An Update

(2010) V. Gieselmann et al.   NEUROPEDIATRICS

Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation

(2009) A Biffi et al.   CLINICAL GENETICS

Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response

(2009) Piotr Hadaczek et al.   HUMAN GENE THERAPY

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

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

(2009) M.-A. Colle et al.   HUMAN MOLECULAR GENETICS

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Efficient Gene Delivery and Selective Transduction of Glial Cells in the Mammalian Brain by AAV Serotypes Isolated From Nonhuman Primates

(2009) Patricia A Lawlor et al.   MOLECULAR THERAPY

Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy

(2008) Tyler Mark Pierson et al.   ANNALS OF NEUROLOGY

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

(2008) Dolan Sondhi et al.   EXPERIMENTAL NEUROLOGY

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY

Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain

(2008) Cassia N Cearley et al.   MOLECULAR THERAPY