All Stories

  1. Neuronal A2A receptor exacerbates synapse loss and memory deficits in APP/PS1 mice
  2. Gene editing as a therapeutic strategy for spinocerebellar ataxia type-3
  3. Mono- and Biallelic Inactivation of Huntingtin Gene in Patient-Specific Induced Pluripotent Stem Cells Reveal HTT Roles in Striatal Development and Neuronal Functions
  4. Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders
  5. Tau Transfer via Extracellular Vesicles Disturbs the Astrocytic Mitochondrial System
  6. Semi-automated workflows to quantify AAV transduction in various brain areas and predict gene editing outcome for neurological disorders
  7. Revisiting the outcome of adult wild-type Htt inactivation in the context of HTT-lowering strategies for Huntington’s disease
  8. Tau promotes oxidative stress-associated cycling neurons in S phase as a pro-survival mechanism: possible implication for Alzheimer’s disease
  9. Disruption of astrocyte-dependent dopamine control in the developing medial prefrontal cortex leads to excessive grooming in mice
  10. Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways
  11. Correction: Maximizing lentiviral vector gene transfer in the CNS
  12. Lactate transporters in the rat barrel cortex sustain whisker-dependent BOLD fMRI signal and behavioral performance
  13. Thrombolysis by PLAT/tPA increases serum free IGF1 leading to a decrease of deleterious autophagy following brain ischemia
  14. Extracellular vesicles: Major actors of heterogeneity in tau spreading among human tauopathies
  15. Corrigendum: Genome Editing for CNS Disorders
  16. Mitochondrial biogenesis in developing astrocytes regulates astrocyte maturation and synapse formation
  17. Astrocytic VMAT2 in the developing prefrontal cortex is required for normal grooming behavior in mice
  18. Tau accumulation in astrocytes of the dentate gyrus induces neuronal dysfunction and memory deficits in Alzheimer’s disease
  19. Genome Editing for CNS Disorders
  20. Glucose metabolism links astroglial mitochondria to cannabinoid effects
  21. Maximizing lentiviral vector gene transfer in the CNS
  22. Efficacy of THN201, a Combination of Donepezil and Mefloquine, to Reverse Neurocognitive Deficits in Alzheimer’s Disease
  23. Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level
  24. Emerging technologies to study glial cells
  25. Genetic and pharmacological inactivation of astroglial connexin 43 differentially influences the acute response of antidepressant and anxiolytic drugs
  26. A New Tool for In Vivo Study of Astrocyte Connexin 43 in Brain
  27. The C-terminal domain of LRRK2 with the G2019S mutation is sufficient to produce neurodegeneration of dopaminergic neurons in vivo
  28. Development of Efficient AAV2/DJ-Based Viral Vectors to Selectively Downregulate the Expression of Neuronal or Astrocytic Target Proteins in the Rat Central Nervous System
  29. Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
  30. Cell-Type-Specific Gene Expression Profiling in Adult Mouse Brain Reveals Normal and Disease-State Signatures
  31. Different tau species lead to heterogeneous tau pathology propagation and misfolding
  32. Therapeutic efficacy of regulable GDNF expression for Huntington's and Parkinson's disease by a high-induction, background-free “GeneSwitch” vector
  33. Human Induced Pluripotent Stem Cell-Derived Astrocytes Are Differentially Activated by Multiple Sclerosis-Associated Cytokines
  34. Dysfunction of homeostatic control of dopamine by astrocytes in the developing prefrontal cortex leads to cognitive impairments
  35. Huntingtin Aggregation Impairs Autophagy, Leading to Argonaute-2 Accumulation and Global MicroRNA Dysregulation
  36. Environment-dependent striatal gene expression in the BACHD rat model for Huntington disease
  37. The striatal kinase DCLK3 produces neuroprotection against mutant huntingtin
  38. CRISPR/Cas9-Mediated Genome Editing for Huntington’s Disease
  39. Scalable Production and Purification of Adeno-Associated Viral Vectors (AAV)
  40. βAPP Processing Drives Gradual Tau Pathology in an Age-Dependent Amyloid Rat Model of Alzheimer’s Disease
  41. The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes
  42. AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease
  43. Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing
  44. A neuronal MCT2 knockdown in the rat somatosensory cortex reduces both the NMR lactate signal and the BOLD response during whisker stimulation
  45. Formation of hippocampal mHTT aggregates leads to impaired spatial memory, hippocampal activation and adult neurogenesis
  46. From huntingtin gene to Huntington’s disease-altering strategies
  47. Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system
  48. Coupling of D2R Short but not D2R Long receptor isoform to the Rho/ROCK signaling pathway renders striatal neurons vulnerable to mutant huntingtin
  49. L5 Pre-clinical evaluation of aav5-mihtt gene therapy of huntington’s disease in rodents
  50. L4 Sustained and strong HTT silencing by AAV5-miHTT as therapy for huntington’s disease
  51. Astrocytes are key but indirect contributors to the development of the symptomatology and pathophysiology of Huntington's disease
  52. 463. Suspension-Adapted HEK 293 Cells in Orbital Shaken Bioreactors for the Production of Adeno-Associated Virus Vectors
  53. 327. Genetic Editing for Huntington's Disease
  54. Dominant-Negative Effects of Adult-Onset Huntingtin Mutations Alter the Division of Human Embryonic Stem Cells-Derived Neural Cells
  55. Alzheimer’s disease-like APP processing in wild-type mice identifies synaptic defects as initial steps of disease progression
  56. Attenuated Levels of Hippocampal Connexin 43 and its Phosphorylation Correlate with Antidepressant- and Anxiolytic-Like Activities in Mice
  57. AMPK activation protects from neuronal dysfunction and vulnerability across nematode, cellular and mouse models of Huntington's disease
  58. Non-human primate model of tauopathy
  59. Gene transfer of both app and ps1 induces hippocampal impairments close to human early phases of Alzheimer’s disease
  60. Gene transfer engineering for astrocyte-specific silencing in the CNS
  61. Microtubule-associated protein 6 mediates neuronal connectivity through Semaphorin 3E-dependent signalling for axonal growth
  62. 465. Transient Production of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Therapy Applications Using Suspension-Adapted HEK 293 Cells in Orbital Shaken Bioreactors
  63. The striatal long noncoding RNA Abhd11os is neuroprotective against an N-terminal fragment of mutant huntingtin in vivo
  64. The JAK/STAT3 Pathway Is a Common Inducer of Astrocyte Reactivity in Alzheimer's and Huntington's Diseases
  65. Approches degene silencingpour le traitement de la maladie de Huntington
  66. Loss of the thyroid hormone-binding protein Crym renders striatal neurons more vulnerable to mutant huntingtin in Huntington's disease
  67. RNA Interference Mitigates Motor and Neuropathological Deficits in a Cerebellar Mouse Model of Machado-Joseph Disease
  68. AMYLOID CASCADE INDUCTION IN AN AAV-BASED MOUSE MODEL OF ALZHEIMER'S DISEASE
  69. Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells
  70. SET translocation is associated with increase in caspase cleaved amyloid precursor protein in CA1 of Alzheimer and Down syndrome patients
  71. Connexin 30 sets synaptic strength by controlling astroglial synapse invasion
  72. Neuron-to-neuron wild-type Tau protein transfer through a trans-synaptic mechanism: relevance to sporadic tauopathies
  73. Lentiviral Vectors in Huntington’s Disease Research and Therapy
  74. Lentiviral Vectors: A Powerful Tool to Target Astrocytes In Vivo
  75. Lentiviral Delivery of the Human Wild-type Tau Protein Mediates a Slow and Progressive Neurodegenerative Tau Pathology in the Rat Brain
  76. In vivo tau spreading relies on the transsynaptic transfer of soluble wild-type tau species
  77. Beclin 1 mitigates motor and neuropathological deficits in genetic mouse models of Machado–Joseph disease
  78. A role of mitochondrial complex II defects in genetic models of Huntington's disease expressing N-terminal fragments of mutant huntingtin
  79. BDNF overexpression in mouse hippocampal astrocytes promotes local neurogenesis and elicits anxiolytic-like activities
  80. Silencing Mutant Ataxin-3 Rescues Motor Deficits and Neuropathology in Machado-Joseph Disease Transgenic Mice
  81. Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors
  82. Lentiviral-Mediated Gene Transfer of siRNAs for the Treatment of Huntington’s Disease
  83. Overexpression of Mutant Ataxin-3 in Mouse Cerebellum Induces Ataxia and Cerebellar Neuropathology
  84. Viral-mediated overexpression of mutant huntingtin to model HD in various species
  85. Restricted transgene expression in the brain with cell-type specific neuronal promoters
  86. Capucin does not modify the toxicity of a mutant Huntingtin fragment in vivo
  87. Restricted Transgene Expression in the Brain with Cell-Type Specific Neuronal Promoters
  88. Calpastatin-mediated inhibition of calpains in the mouse brain prevents mutant ataxin 3 proteolysis, nuclear localization and aggregation, relieving Machado-Joseph disease
  89. Early transcriptional changes linked to naturally occurring Huntington's disease mutations in neural derivatives of human embryonic stem cells
  90. Lentiviral delivery of human wild-type Tau protein mediates a slow and progressive spatio-temporal Tau pathology in rat brain
  91. Nuclear Factor Erythroid 2-Related Factor 2 Facilitates Neuronal Glutathione Synthesis by Upregulating Neuronal Excitatory Amino Acid Transporter 3 Expression
  92. Mitogen- and stress-activated protein kinase 1-induced neuroprotection in Huntington's disease: role on chromatin remodeling at the PGC-1-alpha promoter
  93. Overexpression of the autophagic beclin-1 protein clears mutant ataxin-3 and alleviates Machado–Joseph disease
  94. In vivo expression of polyglutamine-expanded huntingtin by mouse striatal astrocytes impairs glutamate transport: a correlation with Huntington's disease subjects
  95. Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?
  96. Mitochondria in Huntington's disease
  97. Diminished hippocalcin expression in Huntington’s disease brain does not account for increased striatal neuron vulnerability as assessed in primary neurons
  98. Engineered lentiviral vector targeting astrocytesIn vivo
  99. 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
  100. Sustained effects of nonallele-specificHuntingtinsilencing
  101. Normal Aging Modulates the Neurotoxicity of Mutant Huntingtin
  102. Positron Emission Tomography Imaging Demonstrates Correlation between Behavioral Recovery and Correction of Dopamine Neurotransmission after Gene Therapy
  103. Viral Vectors for in Vivo Gene Transfer
  104. Implication of the JNK pathway in a rat model of Huntington's disease
  105. Applications of Lentiviral Vectors for Biology and Gene Therapy of Neurological Disorders
  106. Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease
  107. Gene Transfer Techniques for the Delivery of GDNF in Parkinson's Disease
  108. Dysregulation of Gene Expression in Primary Neuron Models of Huntington's Disease Shows That Polyglutamine-Related Effects on the Striatal Transcriptome May Not Be Dependent on Brain Circuitry
  109. Efficient Gene Transfer and Expression of Biologically Active Glial Cell Line-Derived Neurotrophic Factor in Rat Motoneurons Transduced with Lentiviral Vectors
  110. Vers un nouveau rôle du récepteur PPAR-γ dans le syndrome d’immunodéficience acquise et les hémopathies chez l’homme
  111. Striatal and nigral pathology in a lentiviral rat model of Machado-Joseph disease
  112. Human and simian immunodeficiency viruses deregulate early hematopoiesis through a Nef/PPARγ/STAT5 signaling pathway in macaques
  113. Dopamine determines the vulnerability of striatal neurons to the N-terminal fragment of mutant huntingtin through the regulation of mitochondrial complex II
  114. Haloperidol protects striatal neurons from dysfunction induced by mutated huntingtin in vivo
  115. Expression of Mutated Huntingtin Fragment in the Putamen Is Sufficient to Produce Abnormal Movement in Non-human Primates
  116. Activation of Astrocytes by CNTF Induces Metabolic Plasticity and Increases Resistance to Metabolic Insults
  117. Neuroprotection by Hsp104 and Hsp27 in Lentiviral-based Rat Models of Huntington's Disease
  118. Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts
  119. Transplants of CNTF-producing Cells for the Treatment of Huntington's Disease
  120. Ciliary Neurotrophic Factor Activates Astrocytes, Redistributes Their Glutamate Transporters GLAST and GLT-1 to Raft Microdomains, and Improves Glutamate Handling In Vivo
  121. CA150 Expression Delays Striatal Cell Death in Overexpression and Knock-In Conditions for Mutant Huntingtin Neurotoxicity
  122. Inhibition of Calcineurin by FK506 Protects against Polyglutamine-Huntingtin Toxicity through an Increase of Huntingtin Phosphorylation at S421
  123. Involvement of Mitochondrial Complex II Defects in Neuronal Death Produced by N-Terminus Fragment of Mutated Huntingtin
  124. Lentivirus-mediated expression of glutathione peroxidase: Neuroprotection in murine models of Parkinson's disease
  125. Progressive and selective striatal degeneration in primary neuronal cultures using lentiviral vector coding for a mutant huntingtin fragment
  126. Akt is altered in an animal model of Huntington's disease and in patients
  127. Minocycline in phenotypic models of Huntington's disease
  128. Viral vectors as tools to model and treat neurodegenerative disorders
  129. Lentiviral vector delivery of parkin prevents dopaminergic degeneration in an α-synuclein rat model of Parkinson's disease
  130. Lentiviral nigral delivery of GDNF does not prevent neurodegeneration in a genetic rat model of Parkinson's disease
  131. Neuroprotective Gene Therapy for Huntington's Disease, Using Polymer-Encapsulated Cells Engineered to Secrete Human Ciliary Neurotrophic Factor: Results of a Phase I Study
  132. Survival of Encapsulated Human Primary Fibroblasts and Erythropoietin Expression Under Xenogeneic Conditions
  133. Encapsulated GDNF-producing C2C12 cells for Parkinson's disease: a pre-clinical study in chronic MPTP-treated baboons
  134. Local GDNF expression mediated by lentiviral vector protects facial nerve motoneurons but not spinal motoneurons in SOD1G93A transgenic mice
  135. Long-term lentiviral-mediated expression of ciliary neurotrophic factor in the striatum of Huntington's disease transgenic mice
  136. Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum
  137. Activity analysis of housekeeping promoters using self-inactivating lentiviral vector delivery into the mouse retina
  138. Presence of Gal-alpha1,3Gal epitope on xenogeneic lines: implications for cellular gene therapy based on the encapsulation technology
  139. Multiply Attenuated, Self-Inactivating Lentiviral Vectors Efficiently Deliver and Express Genes for Extended Periods of Time in Adult Rat Cardiomyocytes In Vivo
  140. Optimization of human erythropoietin secretion from MLV-infected human primary fibroblasts used for encapsulated cell therapy
  141. Comparative study of GDNF delivery systems for the CNS: polymer rods, encapsulated cells, and lentiviral vectors
  142. Delivery of Ciliary Neurotrophic Factor via Lentiviral-Mediated Transfer Protects Axotomized Retinal Ganglion Cells for an Extended Period of Time
  143. French gene therapy group reports on the adverse event in a clinical trial of gene therapy for X-linked severe combined immune deficiency (X-SCID)
  144. Lentiviral-Mediated RNA Interference
  145. Dose-Dependent Neuroprotective Effect of Ciliary Neurotrophic Factor Delivered via Tetracycline-Regulated Lentiviral Vectors in the Quinolinic Acid Rat Model of Huntington's Disease
  146. Delivery of erythropoietin by encapsulated myoblasts in a genetic model of severe anemia
  147. Delivery of erythropoietin by encapsulated myoblasts in a genetic model of severe anemia
  148. Neurospheres modified to produce glial cell line-derived neurotrophic factor increase the survival of transplanted dopamine neurons
  149. Long-Term Doxycycline-Regulated Secretion of Erythropoietin by Encapsulated Myoblasts
  150. Seizure Suppression by Adenosine‐releasing Cells Is Independent of Seizure Frequency
  151. α-Synucleinopathy and selective dopaminergic neuron loss in a rat lentiviral-based model of Parkinson's disease
  152. Lentivirally Delivered Glial Cell Line-Derived Neurotrophic Factor Increases the Number of Striatal Dopaminergic Neurons in Primate Models of Nigrostriatal Degeneration
  153. Lentiviral-Mediated Delivery of Mutant Huntingtin in the Striatum of Rats Induces a Selective Neuropathology Modulated by Polyglutamine Repeat Size, Huntingtin Expression Levels, and Protein Length
  154. Lentiviruses as Vectors for CNS Diseases
  155. Neuroprotective effect of interleukin-6 and IL6/IL6R chimera in the quinolinic acid rat model of Huntington's syndrome
  156. Viral vector-mediated gene therapy for Parkinson's disease
  157. Isolation of Multipotent Neural Precursors Residing in the Cortex of the Adult Human Brain
  158. Grafts of adenosine-releasing cells suppress seizures in kindling epilepsy
  159. Neuroprotective Effect of a CNTF-Expressing Lentiviral Vector in the Quinolinic Acid Rat Model of Huntington's Disease
  160. Gene Transfer into Neurons from Hippocampal Slices: Comparison of Recombinant Semliki Forest Virus, Adenovirus, Adeno-Associated Virus, Lentivirus, and Measles Virus
  161. Neurodegeneration Prevented by Lentiviral Vector Delivery of GDNF in Primate Models of Parkinson's Disease
  162. Neuroprotective Gene Therapy for Huntington’s Disease Using a Polymer Encapsulated BHK Cell Line Engineered to Secrete Human CNTF
  163. Complete and Long-Term Rescue of Lesioned Adult Motoneurons by Lentiviral-Mediated Expression of Glial Cell Line-Derived Neurotrophic Factor in the Facial Nucleus
  164. Lentiviral Vectors as a Gene Delivery System in the Mouse Midbrain: Cellular and Behavioral Improvements in a 6-OHDA Model of Parkinson's Disease Using GDNF
  165. Restoration of Cognitive and Motor Functions by Ciliary Neurotrophic Factor in a Primate Model of Huntington's Disease
  166. Self-Inactivating Lentiviral Vectors with Enhanced Transgene Expression as Potential Gene Transfer System in Parkinson's Disease
  167. Lentiviral Gene Transfer to the Nonhuman Primate Brain
  168. Cellular xenotransplantation
  169. Improvement of mouse β-thalassemia upon erythropoietin delivery by encapsulated myoblasts
  170. Continuous delivery of human and mouse erythropoietin in mice by genetically engineered polymer encapsulated myoblasts
  171. A Gene Therapy Approach to Regulated Delivery of Erythropoietin as a Function of Oxygen Tension
  172. A Gene Therapy Approach for the Treatment of Amyotrophic Lateral Sclerosis and Parkinson's Disease
  173. Central Nervous System Delivery of Recombinant Ciliary Neurotrophic Factor by Polymer Encapsulated Differentiated C2C12 Myoblasts
  174. Rescue of motoneurons from axotomy-induced cell death by polymer encapsulated cells genetically engineered to release CNTF
  175. Errata
  176. Continuous delivery of erythropoietin in mice using encapsulated genetically engineered cells lines
  177. Rescue of Motoneurons from Axotomy-Induced Cell Death by Polymer Encapsulated Cells Genetically Engineered to Release CNTF
  178. Intrathecal delivery of CNTF using encapsulated genetically modifiedxenogeneic cells in amyotrophic lateral sclerosis patients
  179. Gene Therapy for Amyotrophic Lateral Sclerosis (ALS) Using a Polymer Encapsulated Xenogenic Cell Line Engineered to Secrete hCNTF. Lausanne University Medical School, Lausanne, Switzerland
  180. Fatty Acids Regulate Thy-1 Antigen mRNA Stability in T Lymphocyte Precursors
  181. Fatty Acids Regulate Thy-1 Antigen mRNA Stability in T Lymphocyte Precursors
  182. Encapsulation of neurotrophic factor-releasing cells for the treatment of neurodegenerative diseases
  183. Minimal membrane and secreted μ poly(A) signals specify developmentally-regulated immunoglobulin heavy chain mRNA ratios without RNA splicing
  184. Membrane μ poly(A) signal and 3′ flanking sequences function as a transcription terminator for immunoglobulin-encoding genes
  185. Multi-level regulation of Thy-1 antigen expression in mouse T lymphomas
  186. Evaluation of somatic cell variants deficient in glycosylphosphatidyl-inositol anchoring as candidates for genetic correction
  187. Isolation from mouse fibroblasts of a cDNA encoding a new form of the fibroblast growth factor receptor (flg)
  188. Translocation of the yeast Dolichol-phosphate-mannose synthase into microsomal membranes
  189. Lentiviral-Mediated Gene Transfer to Model Triplet Repeat Disorders