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

Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB

(2009) Carmela Di Domenico et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Diffusion Tensor Imaging Detects Abnormalities in the Corticospinal Tracts of Neonates with Infantile Krabbe Disease

(2009) M.L. Escolar et al.   AMERICAN JOURNAL OF NEURORADIOLOGY

Delayed post-ischaemic neuroprotection following systemic neural stem cell transplantation involves multiple mechanisms

(2009) Marco Bacigaluppi et al.   BRAIN

Specific Determination of  -Galactocerebrosidase Activity via Competitive Inhibition of  -Galactosidase

(2009) S. Martino et al.   CLINICAL CHEMISTRY

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

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

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Effects of developmental age, brain region, and time in culture on long-term proliferation and multipotency of neural stem cell populations

(2009) Angela Gritti et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Neural precursor cell cultures from GM2 gangliosidosis animal models recapitulate the biochemical and molecular hallmarks of the brain pathology

(2009) Sabata Martino et al.   JOURNAL OF NEUROCHEMISTRY

A Novel and Efficient Gene Transfer Strategy Reduces Glial Reactivity and Improves Neuronal Survival and Axonal Growth In Vitro

(2009) Mathieu Desclaux et al.   PLoS One

Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

(2009) A. P. Kells et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Neural Stem Cell Transplantation Benefits a Monogenic Neurometabolic Disorder During the Symptomatic Phase of Disease

(2009) Mylvaganam Jeyakumar et al.   STEM CELLS

CNS-directed gene therapy for lysosomal storage diseases

(2008) Mark S Sands et al.   ACTA PAEDIATRICA

Clinical Application of Lentiviral Vectors – Concepts and Practice

(2008) Axel Schambach et al.   CURRENT GENE THERAPY

Applications of Lentiviral Vectors for Biology and Gene Therapy of Neurological Disorders

(2008) Cecilia Lundberg et al.   CURRENT GENE THERAPY

Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain

(2008) M L D Broekman et al.   GENE THERAPY

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

Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

(2008) Michael Chang et al.   MOLECULAR THERAPY

Astrocyte-secreted GDNF and glutathione antioxidant system protect neurons against 6OHDA cytotoxicity

(2008) Jagdeep K. Sandhu et al.   NEUROBIOLOGY OF DISEASE