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JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY (2008)
In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
Dirk Grimm et al.
JOURNAL OF VIROLOGY (2008)
Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse
Akihiko Urayama et al.
MOLECULAR THERAPY (2008)
Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis
Michael Chang et al.
MOLECULAR THERAPY (2008)
Preventing growth of brain tumors by creating a zone of resistance
Casey A. Maguire et al.
MOLECULAR THERAPY (2008)
Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain
Cassia N. Cearley et al.
MOLECULAR THERAPY (2008)
Engineering and selection of shuffled AAV genomes: A new strategy for producing targeted biological nanoparticles
Wuping Li et al.
MOLECULAR THERAPY (2008)
DNA shuffling of adeno-associated virus yields functionally diverse viral progeny
James T. Koerber et al.
MOLECULAR THERAPY (2008)
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
Carmela Zincarelli et al.
MOLECULAR THERAPY (2008)
Glioblastoma microvesicles transport RNA and proteins that promote tumour growth and provide diagnostic biomarkers
Johan Skog et al.
NATURE CELL BIOLOGY (2008)
Results from a phase I safety trial of hAADC gene therapy for Parkinson disease
J. L. Eberling et al.
NEUROLOGY (2008)
Subcutaneous IGF-1 is not beneficial in 2-year ALS trial
E. J. Sorenson et al.
NEUROLOGY (2008)
Diffusion in brain extracellular space
Eva Sykova et al.
PHYSIOLOGICAL REVIEWS (2008)
Identification and design of peptides as a new drug delivery system for the brain
Michel Demeule et al.
JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2008)
Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease
Mario A. Cabrera-Salazar et al.
MOLECULAR THERAPY (2007)
A single injection of an Adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease
Cassia N. Cearley et al.
JOURNAL OF NEUROSCIENCE (2007)
Ultrastructure of blood-brain barrier and blood-spinal cord barrier in SOD1 mice modeling ALS
Svitlana Garbuzova-Davis et al.
BRAIN RESEARCH (2007)
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
Michael G. Kaplitt et al.
LANCET (2007)
Translational research models and novel adjunctive therapies for neuroAIDS
M. Christine Zink
JOURNAL OF NEUROIMMUNE PHARMACOLOGY (2007)
Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain
Jean-Marc Taymans et al.
HUMAN GENE THERAPY (2007)
Transduction of brain by herpes simplex virus vectors
Bradford K. Berges et al.
MOLECULAR THERAPY (2007)
Recurrent infections, hypotonia, and mental retardation caused by duplication of MECP2 and adjacent region in Xq28
Michael J. Friez et al.
PEDIATRICS (2006)
Increased MECP2 gene copy number as the result of genomic duplication in neurodevelopmentally delayed males
Daniela del Gaudio et al.
GENETICS IN MEDICINE (2006)
Astrocytic expression of transgene in the rat brain mediated by baculovirus vectors containing an astrocyte-specific promoter
C. Y. Wang et al.
GENE THERAPY (2006)
Blood-brain Barrier: Structural Components and Function Under Physiologic and Pathologic Conditions
Yuri Persidsky et al.
JOURNAL OF NEUROIMMUNE PHARMACOLOGY (2006)
Noninvasive monitoring of long-term lentiviral vector-mediated gene expression in rodent brain with bioluminescence imaging
Christophe M. Deroose et al.
MOLECULAR THERAPY (2006)
Gene therapy of the brain in the dog model of Hurler's syndrome
Carine Ciron et al.
ANNALS OF NEUROLOGY (2006)
A hybrid vector for ligand-directed tumor targeting and molecular imaging
A Hajitou et al.
CELL (2006)
Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice
CD Scallan et al.
BLOOD (2006)
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
CS Manno et al.
NATURE MEDICINE (2006)
Convection-enhanced delivery of adeno-associated virus Type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain
P Hadaczek et al.
HUMAN GENE THERAPY (2006)
Large animal models and gene therapy
M Casal et al.
EUROPEAN JOURNAL OF HUMAN GENETICS (2006)
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
CN Cearley et al.
MOLECULAR THERAPY (2006)
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
N Maheshri et al.
NATURE BIOTECHNOLOGY (2006)
Apolipoprotein E-derived peptides reduce CNS inflammation: implications for therapy of neurological disease
D. T. Laskowitz et al.
ACTA NEUROLOGICA SCANDINAVICA (2006)
Astrocyte-endothelial interactions at the blood-brain barrier
NJ Abbott et al.
NATURE REVIEWS NEUROSCIENCE (2006)
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
GM Acland et al.
MOLECULAR THERAPY (2005)
A segment of the Mecp2 promoter is sufficient to drive expression in neurons
M Adachi et al.
HUMAN MOLECULAR GENETICS (2005)
Effective gene therapy for an inherited CNS disease in a large animal model
CH Vite et al.
ANNALS OF NEUROLOGY (2005)
Rescue and propagation of fully retargeted oncolytic measles viruses
T Nakamura et al.
NATURE BIOTECHNOLOGY (2005)
Differences in transductional tropism of adenoviral and lentiviral vectors in the rat brainstem
H Duale et al.
EXPERIMENTAL PHYSIOLOGY (2005)
Basic fibroblast growth factor enhances transduction, distribution, and axonal transport of adeno-associated virus type 2 vector in rat brain
P Hadaczek et al.
HUMAN GENE THERAPY (2004)
Glioblastoma cells release factors that disrupt blood-brain barrier features
SW Schneider et al.
ACTA NEUROPATHOLOGICA (2004)
Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
C Burger et al.
MOLECULAR THERAPY (2004)
Binding, internalization, and membrane incorporation of human immunodeficiency virus-1 at the blood-brain barrier is differentially regulated
WA Banks et al.
NEUROSCIENCE (2004)
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
SE Raper et al.
MOLECULAR GENETICS AND METABOLISM (2003)
Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of β-glucuronidase-deficient mice
MA Passini et al.
JOURNAL OF VIROLOGY (2003)
Preclinical characterization of the antiglioma activity of a tropism-enhanced adenovirus targeted to the retinoblastoma pathway
J Fueyo et al.
JOURNAL OF THE NATIONAL CANCER INSTITUTE (2003)
Transgene produces massive overexpression of human β-glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors
C Vogler et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2003)
Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area
S Kügler et al.
GENE THERAPY (2003)
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
DM McCarty et al.
GENE THERAPY (2003)
Expression polymorphism of the blood-brain barrier component P-glycoprotein (MDR1) in relation to Parkinson's disease
T Furuno et al.
PHARMACOGENETICS (2002)
Critical issues in gene therapy for neurologic disease
G Hsich et al.
HUMAN GENE THERAPY (2002)
Organ distribution of gene expression after intravenous infusion of targeted and untargeted lentiviral vectors
KW Peng et al.
GENE THERAPY (2001)
A high-efficiency synthetic expression selectively promoter that drives transgene in noradrenergic neurons
DY Hwang et al.
HUMAN GENE THERAPY (2001)
Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes
R Xu et al.
GENE THERAPY (2001)
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
DM McCarty et al.
GENE THERAPY (2001)
Transport of human immunodeficiency virus type 1 pseudoviruses across the blood-brain barrier: Role of envelope proteins and adsorptive endocytosis
WA Banks et al.
JOURNAL OF VIROLOGY (2001)
Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain
CE Thomas et al.
MOLECULAR THERAPY (2001)
Sequestration of adenoviral vector by Kupffer cells leads to a nonlinear dose response of transduction in liver
NJ Tao et al.
MOLECULAR THERAPY (2001)
Breeding of retroviruses by DNA shuffling for improved stability and processing yields
SK Powell et al.
NATURE BIOTECHNOLOGY (2000)
Neural stem cells display extensive tropism for pathology in adult brain: Evidence from intracranial gliomas
KS Aboody et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)
Glial cell line-derived neurotrophic factor (GDNF) as a defensive molecule for neurodegenerative disease: a tribute to the studies of Antonia Vernadakis on neuronal-glial interactions
MC Bohn et al.
INTERNATIONAL JOURNAL OF DEVELOPMENTAL NEUROSCIENCE (2000)
Pretreatment with protease is a useful experimental strategy for enhancing adenovirus-mediated cancer gene therapy
N Kuriyama et al.
HUMAN GENE THERAPY (2000)
Hyaluronidase enhances recombinant adeno-associated virus (rAAV)-mediated gene transfer in the rat skeletal muscle
D Favre et al.
GENE THERAPY (2000)
Novel transcriptional regulatory signals in the adeno-associated virus terminal repeat A/D junction element
RP Haberman et al.
JOURNAL OF VIROLOGY (2000)
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