Related references
Note: Only part of the references are listed.Reversibility of neuropathology in Tay-Sachs-related diseases
Maria-Begona Cachon-Gonzalez et al.
HUMAN MOLECULAR GENETICS (2014)
Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10
Bin Yang et al.
MOLECULAR THERAPY (2014)
Sustained Normalization of Neurological Disease After Intracranial Gene Therapy in a Feline Model
Victoria J. McCurdy et al.
SCIENCE TRANSLATIONAL MEDICINE (2014)
Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF
Yoshiyuki Yamazaki et al.
SCIENTIFIC REPORTS (2014)
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
Virginia Haurigot et al.
JOURNAL OF CLINICAL INVESTIGATION (2013)
Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy
Allison M. Bradbury et al.
MOLECULAR THERAPY (2013)
Ethylenedioxy-PIP2 Oxalate Reduces Ganglioside Storage in Juvenile Sandhoff Disease Mice
Julian R. Arthur et al.
NEUROCHEMICAL RESEARCH (2013)
Deep Cortical Layers Are Activated Directly by Thalamus
Christine M. Constantinople et al.
SCIENCE (2013)
Gene Transfer Corrects Acute GM2 Gangliosidosis-Potential Therapeutic Contribution of Perivascular Enzyme Flow
M. Begona Cachon-Gonzalez et al.
MOLECULAR THERAPY (2012)
Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain
Timothy J. Sargeant et al.
HUMAN MOLECULAR GENETICS (2011)
AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival
Rena C. Baek et al.
PLOS ONE (2010)
Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain
M. L. D. Broekman et al.
GENE THERAPY (2009)
Neurodegenerative lysosomal storage disease in European Burmese cats with hexosaminidase β-subunit deficiency
Allison M. Bradbury et al.
MOLECULAR GENETICS AND METABOLISM (2009)
Comparative Analysis of Brain Lipids in Mice, Cats, and Humans with Sandhoff Disease
Rena C. Baek et al.
LIPIDS (2009)
N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice
Rena C. Baek et al.
NEUROCHEMISTRY INTERNATIONAL (2008)
Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease
Jean-Pyo Lee et al.
NATURE MEDICINE (2007)
Complete correction of enzymatic deficiency and neurochemistry in the GM1-gangliosidosis mouse brain by neonatal adeno-associated virus-mediated gene delivery
M. L. D. Broekman et al.
MOLECULAR THERAPY (2007)
Effective gene therapy in an authentic model of Tay-Sachs-related diseases
M. Begona Cachon-Gonzalez et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Influence of caloric restriction on motor behavior, longevity, and brain lipid composition in Sandhoff disease mice
CA Denny et al.
JOURNAL OF NEUROSCIENCE RESEARCH (2006)
Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins
RL Klein et al.
MOLECULAR THERAPY (2006)
Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors
GM Liu et al.
JOURNAL OF NEUROSCIENCE (2005)
Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice
JL Kasperzyk et al.
JOURNAL OF LIPID RESEARCH (2005)
N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis
JL Kasperzyk et al.
JOURNAL OF NEUROCHEMISTRY (2004)
Inheritance of lysosomal acid β-galactosidase activity and gangliosides in crosses of DBA/2J and knockout mice
EC Hauser et al.
BIOCHEMICAL GENETICS (2004)
An inversion of 25 base pairs causes feline GM2 gangliosidosis variant 0
DR Martin et al.
EXPERIMENTAL NEUROLOGY (2004)
Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for Canavan disease
R Matalon et al.
MOLECULAR THERAPY (2003)
Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis
M Jeyakumar et al.
BRAIN (2003)
Aberrant sprouting and downregulation of tyrosine hydroxylase in lesioned nigrostriatal dopamine neurons induced by long-lasting overexpression of glial cell line derived neurotrophic factor in the striatum by lentiviral gene transfer
B Georgievska et al.
EXPERIMENTAL NEUROLOGY (2002)