相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease
Heather L. Gray-Edwards et al.
HUMAN GENE THERAPY (2018)
Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice
Natalia Niemir et al.
HUMAN MOLECULAR GENETICS (2018)
Molecular Diversity and Specializations among the Cells of the Adult Mouse Brain
Arpiar Saunders et al.
CELL (2018)
A molecular atlas of cell types and zonation in the brain vasculature
Michael Vanlandewijck et al.
NATURE (2018)
Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain
Diane Golebiowski et al.
HUMAN GENE THERAPY (2017)
AAV-MEDIATED GENE DELIVERY ATTENUATES NEUROINFLAMMATION IN FELINE SANDHOFF DISEASE
Allison M. Bradbury et al.
NEUROSCIENCE (2017)
Brain microvasculature defects and Glut1 deficiency syndrome averted by early repletion of the glucose transporter-1 protein
Maoxue Tang et al.
NATURE COMMUNICATIONS (2017)
Gene Therapy Decreases Seizures in a Model of Incontinentia pigmenti
Godwin K. Dogbevia et al.
ANNALS OF NEUROLOGY (2017)
Modeling Psychomotor Retardation using iPSCs from MCT8-Deficient Patients Indicates a Prominent Role for the Blood-Brain Barrier
Gad D. Vatine et al.
CELL STEM CELL (2017)
A brain microvasculature endothelial cell-specific viral vector with the potential to treat neurovascular and neurological diseases
Jakob Koerbelin et al.
EMBO MOLECULAR MEDICINE (2016)
Comprehensive untargeted lipidomic analysis using core-shell C30 particle column and high field orbitrap mass spectrometer
Monica Narvaez-Rivas et al.
JOURNAL OF CHROMATOGRAPHY A (2016)
Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model
Keisuke Kitakaze et al.
JOURNAL OF CLINICAL INVESTIGATION (2016)
Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo
Michael B. Tropak et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)
Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates
Jagdeep S. Walia et al.
MOLECULAR THERAPY (2015)
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
Jean-Charles Nault et al.
NATURE GENETICS (2015)
A new liquid chromatography/tandem mass spectrometry method for quantification of gangliosides in human plasma
Qianyang Huang et al.
ANALYTICAL BIOCHEMISTRY (2014)
Deletion of tumor necrosis factor-α ameliorates neurodegeneration in Sandhoff disease mice
Hatem Abo-Ouf et al.
HUMAN MOLECULAR GENETICS (2013)
In vivo imaging of cerebral microvascular plasticity from birth to death
Roa Harb et al.
JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM (2013)
Gangliosides and Gangliosidoses: Principles of Molecular and Metabolic Pathogenesis
Konrad Sandhoff et al.
JOURNAL OF NEUROSCIENCE (2013)
The microglial sensome revealed by direct RNA sequencing
Suzanne E. Hickman et al.
NATURE NEUROSCIENCE (2013)
Conditional expression of human β-hexosaminidase in the neurons of Sandhoff disease rescues mice from neurodegeneration but not neuroinflammation
Stephanos Kyrkanides et al.
JOURNAL OF NEUROINFLAMMATION (2012)
Gene Transfer Corrects Acute GM2 Gangliosidosis-Potential Therapeutic Contribution of Perivascular Enzyme Flow
M. Begona Cachon-Gonzalez et al.
MOLECULAR THERAPY (2012)
Highly Phosphomannosylated Enzyme Replacement Therapy for GM2 Gangliosidosis
Daisuke Tsuji et al.
ANNALS OF NEUROLOGY (2011)
TAK1 in brain endothelial cells mediates fever and lethargy
Dirk A. Ridder et al.
JOURNAL OF EXPERIMENTAL MEDICINE (2011)
Induced secretion of β-hexosaminidase by human brain endothelial cells: A novel approach in Sandhoff disease?
Lionel Batista et al.
NEUROBIOLOGY OF DISEASE (2010)
Hexosaminidase assays
Michaela Wendeler et al.
GLYCOCONJUGATE JOURNAL (2009)
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Kevin D. Foust et al.
NATURE BIOTECHNOLOGY (2009)
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
Yong Hong Chen et al.
NATURE MEDICINE (2009)
Toxicity from different SOD1 mutants dysregulates the complement system and the neuronal regenerative response in ALS motor neurons
Christian S. Lobsiger et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6
D Grimm et al.
MOLECULAR THERAPY (2003)