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Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

Nature neuroscience, 2021-07, Vol.24 (7), p.930-940

2021

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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Neuron (Cambridge, Mass.), 2012-06, Vol.74 (6), p.1031-1044

2012

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Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

Proceedings of the National Academy of Sciences - PNAS, 2017-03, Vol.114 (10), p.2699-2704

2017

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Dysregulation of Mdm2 and Mdm4 alternative splicing underlies motor neuron death in spinal muscular atrophy

Genes & development, 2018-08, Vol.32 (15-16), p.1045-1059

2018

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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

Science translational medicine, 2011-03, Vol.3 (72), p.72ra18

2011

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Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies

Proceedings of the National Academy of Sciences - PNAS, 2013-02, Vol.110 (9), p.3537-3542

2013

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Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis

Proceedings of the National Academy of Sciences - PNAS, 2015-06, Vol.112 (26), p.8100-8105

2015

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Neutral Lipid Cacostasis Contributes to Disease Pathogenesis in Amyotrophic Lateral Sclerosis

The Journal of neuroscience, 2020-11, Vol.40 (47), p.9137-9147

2020

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CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy

Proceedings of the National Academy of Sciences - PNAS, 2011-07, Vol.108 (29), p.12101-12106

2011

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Astrocyte transduction is required for rescue of behavioral phenotypes in the YAC128 mouse model with AAV-RNAi mediated HTT lowering therapeutics

Neurobiology of disease, 2019-09, Vol.129, p.29-37

2019

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Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model

Scientific reports, 2021-10, Vol.11 (1), p.20945-20945, Article 20945

2021

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Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis

Proceedings of the National Academy of Sciences - PNAS, 2013-06, Vol.110 (26), p.10812-10817

2013

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Stem cell transplantation for neurometabolic and neurodegenerative diseases

Neuropharmacology, 2010-05, Vol.58 (6), p.845-854

2010

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Viral delivery of a microRNA to Gba to the mouse central nervous system models neuronopathic Gaucher disease

Neurobiology of disease, 2019-10, Vol.130, p.104513-104513, Article 104513

2019

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Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy

Human gene therapy, 2014-07, Vol.25 (7), p.619-630

2014

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Adult Spinal Cord Stem Cells Generate Neurons after Transplantation in the Adult Dentate Gyrus

The Journal of neuroscience, 2000-12, Vol.20 (23), p.8727-8735

2000

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AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

Molecular therapy, 2010-12, Vol.18 (12), p.2075-2084

2010

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Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer's disease

Alzheimer's research & therapy, 2022-02, Vol.14 (1), p.19-19, Article 19

2022

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Partial rescue of some features of Huntington Disease in the genetic absence of caspase-6 in YAC128 mice

Neurobiology of disease, 2015-04, Vol.76, p.24-36

2015

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Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity

Molecular therapy, 2008-06, Vol.16 (6), p.1056-1064

2008

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Neural Stem Cell Transplantation as a Therapeutic Approach for Treating Lysosomal Storage Diseases

Neurotherapeutics, 2011-10, Vol.8 (4), p.659-667

2011

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Polysialic acid regulates the clustering, migration, and neuronal differentiation of progenitor cells in the adult hippocampus

Developmental neurobiology (Hoboken, N.J.), 2008-12, Vol.68 (14), p.1580-1590

2008

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New Frontiers in Parkinson's Disease: From Genetics to the Clinic

The Journal of neuroscience, 2018-10, Vol.38 (44), p.9375-9382

2018

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Acid β-glucosidase mutants linked to gaucher disease, parkinson disease, and lewy body dementia alter α-synuclein processing

Annals of neurology, 2011-06, Vol.69 (6), p.940-953

2011

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Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease

Human gene therapy, 2014-05, Vol.25 (5), p.461-474

2014

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Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

Dysregulation of Mdm2 and Mdm4 alternative splicing underlies motor neuron death in spinal muscular atrophy

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies

Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis

Neutral Lipid Cacostasis Contributes to Disease Pathogenesis in Amyotrophic Lateral Sclerosis

CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy

Astrocyte transduction is required for rescue of behavioral phenotypes in the YAC128 mouse model with AAV-RNAi mediated HTT lowering therapeutics

Preclinical pharmacology of glucosylceramide synthase inhibitor venglustat in a GBA-related synucleinopathy model

Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis

Stem cell transplantation for neurometabolic and neurodegenerative diseases

Viral delivery of a microRNA to Gba to the mouse central nervous system models neuronopathic Gaucher disease

Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy

Adult Spinal Cord Stem Cells Generate Neurons after Transplantation in the Adult Dentate Gyrus

AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer's disease

Partial rescue of some features of Huntington Disease in the genetic absence of caspase-6 in YAC128 mice

Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity

Neural Stem Cell Transplantation as a Therapeutic Approach for Treating Lysosomal Storage Diseases

Polysialic acid regulates the clustering, migration, and neuronal differentiation of progenitor cells in the adult hippocampus

New Frontiers in Parkinson's Disease: From Genetics to the Clinic

Acid β-glucosidase mutants linked to gaucher disease, parkinson disease, and lewy body dementia alter α-synuclein processing

Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease

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