All Stories

  1. Selective striatal pathological changes in a novel human HTT exon 1 knock-in mouse model of Huntington's disease
  2. Gene editing for Spinocerebellar ataxia type 3 taking advantage of the human ATXN3L paralog as replacement gene
  3. Extracellular PHF-tau modulates astrocyte mitochondrial dynamics and mediates neuronal connectivity
  4. Plasminogen activator inhibitor-1 regulates Zika virus infection
  5. Neuronal A2A receptor exacerbates synapse loss and memory deficits in APP/PS1 mice
  6. Gene editing as a therapeutic strategy for spinocerebellar ataxia type-3
  7. Mono- and Biallelic Inactivation of Huntingtin Gene in Patient-Specific Induced Pluripotent Stem Cells Reveal HTT Roles in Striatal Development and Neuronal Functions
  8. Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders
  9. Tau Transfer via Extracellular Vesicles Disturbs the Astrocytic Mitochondrial System
  10. Semi-automated workflows to quantify AAV transduction in various brain areas and predict gene editing outcome for neurological disorders
  11. Revisiting the outcome of adult wild-type Htt inactivation in the context of HTT-lowering strategies for Huntington’s disease
  12. Tau promotes oxidative stress-associated cycling neurons in S phase as a pro-survival mechanism: possible implication for Alzheimer’s disease
  13. Disruption of astrocyte-dependent dopamine control in the developing medial prefrontal cortex leads to excessive grooming in mice
  14. Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways
  15. Correction: Maximizing lentiviral vector gene transfer in the CNS
  16. Lactate transporters in the rat barrel cortex sustain whisker-dependent BOLD fMRI signal and behavioral performance
  17. Thrombolysis by PLAT/tPA increases serum free IGF1 leading to a decrease of deleterious autophagy following brain ischemia
  18. Extracellular vesicles: Major actors of heterogeneity in tau spreading among human tauopathies
  19. Corrigendum: Genome Editing for CNS Disorders
  20. Mitochondrial biogenesis in developing astrocytes regulates astrocyte maturation and synapse formation
  21. Astrocytic VMAT2 in the developing prefrontal cortex is required for normal grooming behavior in mice
  22. Tau accumulation in astrocytes of the dentate gyrus induces neuronal dysfunction and memory deficits in Alzheimer’s disease
  23. Genome Editing for CNS Disorders
  24. Glucose metabolism links astroglial mitochondria to cannabinoid effects
  25. Maximizing lentiviral vector gene transfer in the CNS
  26. Efficacy of THN201, a Combination of Donepezil and Mefloquine, to Reverse Neurocognitive Deficits in Alzheimer’s Disease
  27. Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level
  28. Emerging technologies to study glial cells
  29. Genetic and pharmacological inactivation of astroglial connexin 43 differentially influences the acute response of antidepressant and anxiolytic drugs
  30. A New Tool for In Vivo Study of Astrocyte Connexin 43 in Brain
  31. The C-terminal domain of LRRK2 with the G2019S mutation is sufficient to produce neurodegeneration of dopaminergic neurons in vivo
  32. 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
  33. Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
  34. Cell-Type-Specific Gene Expression Profiling in Adult Mouse Brain Reveals Normal and Disease-State Signatures
  35. Different tau species lead to heterogeneous tau pathology propagation and misfolding
  36. Therapeutic efficacy of regulable GDNF expression for Huntington's and Parkinson's disease by a high-induction, background-free “GeneSwitch” vector
  37. Human Induced Pluripotent Stem Cell-Derived Astrocytes Are Differentially Activated by Multiple Sclerosis-Associated Cytokines
  38. Dysfunction of homeostatic control of dopamine by astrocytes in the developing prefrontal cortex leads to cognitive impairments
  39. Huntingtin Aggregation Impairs Autophagy, Leading to Argonaute-2 Accumulation and Global MicroRNA Dysregulation
  40. Environment-dependent striatal gene expression in the BACHD rat model for Huntington disease
  41. The striatal kinase DCLK3 produces neuroprotection against mutant huntingtin
  42. CRISPR/Cas9-Mediated Genome Editing for Huntington’s Disease
  43. Scalable Production and Purification of Adeno-Associated Viral Vectors (AAV)
  44. βAPP Processing Drives Gradual Tau Pathology in an Age-Dependent Amyloid Rat Model of Alzheimer’s Disease
  45. The Self-Inactivating KamiCas9 System for the Editing of CNS Disease Genes
  46. AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease
  47. Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing
  48. A neuronal MCT2 knockdown in the rat somatosensory cortex reduces both the NMR lactate signal and the BOLD response during whisker stimulation
  49. Formation of hippocampal mHTT aggregates leads to impaired spatial memory, hippocampal activation and adult neurogenesis
  50. From huntingtin gene to Huntington’s disease-altering strategies
  51. Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system
  52. Coupling of D2R Short but not D2R Long receptor isoform to the Rho/ROCK signaling pathway renders striatal neurons vulnerable to mutant huntingtin
  53. L5 Pre-clinical evaluation of aav5-mihtt gene therapy of huntington’s disease in rodents
  54. L4 Sustained and strong HTT silencing by AAV5-miHTT as therapy for huntington’s disease
  55. Astrocytes are key but indirect contributors to the development of the symptomatology and pathophysiology of Huntington's disease
  56. 463. Suspension-Adapted HEK 293 Cells in Orbital Shaken Bioreactors for the Production of Adeno-Associated Virus Vectors
  57. 327. Genetic Editing for Huntington's Disease
  58. Dominant-Negative Effects of Adult-Onset Huntingtin Mutations Alter the Division of Human Embryonic Stem Cells-Derived Neural Cells
  59. Alzheimer’s disease-like APP processing in wild-type mice identifies synaptic defects as initial steps of disease progression
  60. Attenuated Levels of Hippocampal Connexin 43 and its Phosphorylation Correlate with Antidepressant- and Anxiolytic-Like Activities in Mice
  61. AMPK activation protects from neuronal dysfunction and vulnerability across nematode, cellular and mouse models of Huntington's disease
  62. Non-human primate model of tauopathy
  63. Gene transfer of both app and ps1 induces hippocampal impairments close to human early phases of Alzheimer’s disease
  64. Gene transfer engineering for astrocyte-specific silencing in the CNS
  65. Microtubule-associated protein 6 mediates neuronal connectivity through Semaphorin 3E-dependent signalling for axonal growth
  66. 465. Transient Production of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Therapy Applications Using Suspension-Adapted HEK 293 Cells in Orbital Shaken Bioreactors
  67. The striatal long noncoding RNA Abhd11os is neuroprotective against an N-terminal fragment of mutant huntingtin in vivo
  68. The JAK/STAT3 Pathway Is a Common Inducer of Astrocyte Reactivity in Alzheimer's and Huntington's Diseases
  69. Approches degene silencingpour le traitement de la maladie de Huntington
  70. Loss of the thyroid hormone-binding protein Crym renders striatal neurons more vulnerable to mutant huntingtin in Huntington's disease
  71. RNA Interference Mitigates Motor and Neuropathological Deficits in a Cerebellar Mouse Model of Machado-Joseph Disease
  72. AMYLOID CASCADE INDUCTION IN AN AAV-BASED MOUSE MODEL OF ALZHEIMER'S DISEASE
  73. Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells
  74. SET translocation is associated with increase in caspase cleaved amyloid precursor protein in CA1 of Alzheimer and Down syndrome patients
  75. Connexin 30 sets synaptic strength by controlling astroglial synapse invasion
  76. Neuron-to-neuron wild-type Tau protein transfer through a trans-synaptic mechanism: relevance to sporadic tauopathies
  77. Lentiviral Vectors in Huntington’s Disease Research and Therapy
  78. Lentiviral Vectors: A Powerful Tool to Target Astrocytes In Vivo
  79. Lentiviral Delivery of the Human Wild-type Tau Protein Mediates a Slow and Progressive Neurodegenerative Tau Pathology in the Rat Brain
  80. In vivo tau spreading relies on the transsynaptic transfer of soluble wild-type tau species
  81. Beclin 1 mitigates motor and neuropathological deficits in genetic mouse models of Machado–Joseph disease
  82. A role of mitochondrial complex II defects in genetic models of Huntington's disease expressing N-terminal fragments of mutant huntingtin
  83. BDNF overexpression in mouse hippocampal astrocytes promotes local neurogenesis and elicits anxiolytic-like activities
  84. Silencing Mutant Ataxin-3 Rescues Motor Deficits and Neuropathology in Machado-Joseph Disease Transgenic Mice
  85. Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors
  86. Lentiviral-Mediated Gene Transfer of siRNAs for the Treatment of Huntington’s Disease
  87. Overexpression of Mutant Ataxin-3 in Mouse Cerebellum Induces Ataxia and Cerebellar Neuropathology
  88. Viral-mediated overexpression of mutant huntingtin to model HD in various species
  89. Restricted transgene expression in the brain with cell-type specific neuronal promoters
  90. Capucin does not modify the toxicity of a mutant Huntingtin fragment in vivo
  91. Restricted Transgene Expression in the Brain with Cell-Type Specific Neuronal Promoters
  92. Calpastatin-mediated inhibition of calpains in the mouse brain prevents mutant ataxin 3 proteolysis, nuclear localization and aggregation, relieving Machado-Joseph disease
  93. Early transcriptional changes linked to naturally occurring Huntington's disease mutations in neural derivatives of human embryonic stem cells
  94. Lentiviral delivery of human wild-type Tau protein mediates a slow and progressive spatio-temporal Tau pathology in rat brain
  95. Nuclear Factor Erythroid 2-Related Factor 2 Facilitates Neuronal Glutathione Synthesis by Upregulating Neuronal Excitatory Amino Acid Transporter 3 Expression
  96. Mitogen- and stress-activated protein kinase 1-induced neuroprotection in Huntington's disease: role on chromatin remodeling at the PGC-1-alpha promoter
  97. Overexpression of the autophagic beclin-1 protein clears mutant ataxin-3 and alleviates Machado–Joseph disease
  98. In vivo expression of polyglutamine-expanded huntingtin by mouse striatal astrocytes impairs glutamate transport: a correlation with Huntington's disease subjects
  99. Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?
  100. Mitochondria in Huntington's disease
  101. Diminished hippocalcin expression in Huntington’s disease brain does not account for increased striatal neuron vulnerability as assessed in primary neurons
  102. Engineered lentiviral vector targeting astrocytesIn vivo
  103. 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
  104. Sustained effects of nonallele-specificHuntingtinsilencing
  105. Normal Aging Modulates the Neurotoxicity of Mutant Huntingtin
  106. Positron Emission Tomography Imaging Demonstrates Correlation between Behavioral Recovery and Correction of Dopamine Neurotransmission after Gene Therapy
  107. Viral Vectors for in Vivo Gene Transfer
  108. Implication of the JNK pathway in a rat model of Huntington's disease
  109. Applications of Lentiviral Vectors for Biology and Gene Therapy of Neurological Disorders
  110. Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease
  111. Gene Transfer Techniques for the Delivery of GDNF in Parkinson's Disease
  112. 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
  113. Efficient Gene Transfer and Expression of Biologically Active Glial Cell Line-Derived Neurotrophic Factor in Rat Motoneurons Transduced with Lentiviral Vectors
  114. Vers un nouveau rôle du récepteur PPAR-γ dans le syndrome d’immunodéficience acquise et les hémopathies chez l’homme
  115. Striatal and nigral pathology in a lentiviral rat model of Machado-Joseph disease
  116. Human and simian immunodeficiency viruses deregulate early hematopoiesis through a Nef/PPARγ/STAT5 signaling pathway in macaques
  117. Dopamine determines the vulnerability of striatal neurons to the N-terminal fragment of mutant huntingtin through the regulation of mitochondrial complex II
  118. Haloperidol protects striatal neurons from dysfunction induced by mutated huntingtin in vivo
  119. Expression of Mutated Huntingtin Fragment in the Putamen Is Sufficient to Produce Abnormal Movement in Non-human Primates
  120. Activation of Astrocytes by CNTF Induces Metabolic Plasticity and Increases Resistance to Metabolic Insults
  121. Neuroprotection by Hsp104 and Hsp27 in Lentiviral-based Rat Models of Huntington's Disease
  122. Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts
  123. Transplants of CNTF-producing Cells for the Treatment of Huntington's Disease
  124. Ciliary Neurotrophic Factor Activates Astrocytes, Redistributes Their Glutamate Transporters GLAST and GLT-1 to Raft Microdomains, and Improves Glutamate Handling In Vivo
  125. CA150 Expression Delays Striatal Cell Death in Overexpression and Knock-In Conditions for Mutant Huntingtin Neurotoxicity
  126. Inhibition of Calcineurin by FK506 Protects against Polyglutamine-Huntingtin Toxicity through an Increase of Huntingtin Phosphorylation at S421
  127. Involvement of Mitochondrial Complex II Defects in Neuronal Death Produced by N-Terminus Fragment of Mutated Huntingtin
  128. Lentivirus-mediated expression of glutathione peroxidase: Neuroprotection in murine models of Parkinson's disease
  129. Progressive and selective striatal degeneration in primary neuronal cultures using lentiviral vector coding for a mutant huntingtin fragment
  130. Akt is altered in an animal model of Huntington's disease and in patients
  131. Minocycline in phenotypic models of Huntington's disease
  132. Viral vectors as tools to model and treat neurodegenerative disorders
  133. Lentiviral vector delivery of parkin prevents dopaminergic degeneration in an α-synuclein rat model of Parkinson's disease
  134. Lentiviral nigral delivery of GDNF does not prevent neurodegeneration in a genetic rat model of Parkinson's disease
  135. Neuroprotective Gene Therapy for Huntington's Disease, Using Polymer-Encapsulated Cells Engineered to Secrete Human Ciliary Neurotrophic Factor: Results of a Phase I Study
  136. Survival of Encapsulated Human Primary Fibroblasts and Erythropoietin Expression Under Xenogeneic Conditions
  137. Encapsulated GDNF-producing C2C12 cells for Parkinson's disease: a pre-clinical study in chronic MPTP-treated baboons
  138. Local GDNF expression mediated by lentiviral vector protects facial nerve motoneurons but not spinal motoneurons in SOD1G93A transgenic mice
  139. Long-term lentiviral-mediated expression of ciliary neurotrophic factor in the striatum of Huntington's disease transgenic mice
  140. Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum
  141. Activity analysis of housekeeping promoters using self-inactivating lentiviral vector delivery into the mouse retina
  142. Presence of Gal-alpha1,3Gal epitope on xenogeneic lines: implications for cellular gene therapy based on the encapsulation technology
  143. Multiply Attenuated, Self-Inactivating Lentiviral Vectors Efficiently Deliver and Express Genes for Extended Periods of Time in Adult Rat Cardiomyocytes In Vivo
  144. Optimization of human erythropoietin secretion from MLV-infected human primary fibroblasts used for encapsulated cell therapy
  145. Comparative study of GDNF delivery systems for the CNS: polymer rods, encapsulated cells, and lentiviral vectors
  146. Delivery of Ciliary Neurotrophic Factor via Lentiviral-Mediated Transfer Protects Axotomized Retinal Ganglion Cells for an Extended Period of Time
  147. 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)
  148. Lentiviral-Mediated RNA Interference
  149. Dose-Dependent Neuroprotective Effect of Ciliary Neurotrophic Factor Delivered via Tetracycline-Regulated Lentiviral Vectors in the Quinolinic Acid Rat Model of Huntington's Disease
  150. Delivery of erythropoietin by encapsulated myoblasts in a genetic model of severe anemia
  151. Delivery of erythropoietin by encapsulated myoblasts in a genetic model of severe anemia
  152. Neurospheres modified to produce glial cell line-derived neurotrophic factor increase the survival of transplanted dopamine neurons
  153. Long-Term Doxycycline-Regulated Secretion of Erythropoietin by Encapsulated Myoblasts
  154. Seizure Suppression by Adenosine‐releasing Cells Is Independent of Seizure Frequency
  155. α-Synucleinopathy and selective dopaminergic neuron loss in a rat lentiviral-based model of Parkinson's disease
  156. Lentivirally Delivered Glial Cell Line-Derived Neurotrophic Factor Increases the Number of Striatal Dopaminergic Neurons in Primate Models of Nigrostriatal Degeneration
  157. 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
  158. Lentiviruses as Vectors for CNS Diseases
  159. Neuroprotective effect of interleukin-6 and IL6/IL6R chimera in the quinolinic acid rat model of Huntington's syndrome
  160. Viral vector-mediated gene therapy for Parkinson's disease
  161. Isolation of Multipotent Neural Precursors Residing in the Cortex of the Adult Human Brain
  162. Grafts of adenosine-releasing cells suppress seizures in kindling epilepsy
  163. Neuroprotective Effect of a CNTF-Expressing Lentiviral Vector in the Quinolinic Acid Rat Model of Huntington's Disease
  164. Gene Transfer into Neurons from Hippocampal Slices: Comparison of Recombinant Semliki Forest Virus, Adenovirus, Adeno-Associated Virus, Lentivirus, and Measles Virus
  165. Neurodegeneration Prevented by Lentiviral Vector Delivery of GDNF in Primate Models of Parkinson's Disease
  166. Neuroprotective Gene Therapy for Huntington’s Disease Using a Polymer Encapsulated BHK Cell Line Engineered to Secrete Human CNTF
  167. Complete and Long-Term Rescue of Lesioned Adult Motoneurons by Lentiviral-Mediated Expression of Glial Cell Line-Derived Neurotrophic Factor in the Facial Nucleus
  168. 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
  169. Restoration of Cognitive and Motor Functions by Ciliary Neurotrophic Factor in a Primate Model of Huntington's Disease
  170. Self-Inactivating Lentiviral Vectors with Enhanced Transgene Expression as Potential Gene Transfer System in Parkinson's Disease
  171. Lentiviral Gene Transfer to the Nonhuman Primate Brain
  172. Cellular xenotransplantation
  173. Improvement of mouse β-thalassemia upon erythropoietin delivery by encapsulated myoblasts
  174. Continuous delivery of human and mouse erythropoietin in mice by genetically engineered polymer encapsulated myoblasts
  175. A Gene Therapy Approach to Regulated Delivery of Erythropoietin as a Function of Oxygen Tension
  176. A Gene Therapy Approach for the Treatment of Amyotrophic Lateral Sclerosis and Parkinson's Disease
  177. Central Nervous System Delivery of Recombinant Ciliary Neurotrophic Factor by Polymer Encapsulated Differentiated C2C12 Myoblasts
  178. Rescue of motoneurons from axotomy-induced cell death by polymer encapsulated cells genetically engineered to release CNTF
  179. Errata
  180. Continuous delivery of erythropoietin in mice using encapsulated genetically engineered cells lines
  181. Rescue of Motoneurons from Axotomy-Induced Cell Death by Polymer Encapsulated Cells Genetically Engineered to Release CNTF
  182. Intrathecal delivery of CNTF using encapsulated genetically modifiedxenogeneic cells in amyotrophic lateral sclerosis patients
  183. Gene Therapy for Amyotrophic Lateral Sclerosis (ALS) Using a Polymer Encapsulated Xenogenic Cell Line Engineered to Secrete hCNTF. Lausanne University Medical School, Lausanne, Switzerland
  184. Fatty Acids Regulate Thy-1 Antigen mRNA Stability in T Lymphocyte Precursors
  185. Fatty Acids Regulate Thy-1 Antigen mRNA Stability in T Lymphocyte Precursors
  186. Encapsulation of neurotrophic factor-releasing cells for the treatment of neurodegenerative diseases
  187. Minimal membrane and secreted μ poly(A) signals specify developmentally-regulated immunoglobulin heavy chain mRNA ratios without RNA splicing
  188. Membrane μ poly(A) signal and 3′ flanking sequences function as a transcription terminator for immunoglobulin-encoding genes
  189. Multi-level regulation of Thy-1 antigen expression in mouse T lymphomas
  190. Evaluation of somatic cell variants deficient in glycosylphosphatidyl-inositol anchoring as candidates for genetic correction
  191. Isolation from mouse fibroblasts of a cDNA encoding a new form of the fibroblast growth factor receptor (flg)
  192. Translocation of the yeast Dolichol-phosphate-mannose synthase into microsomal membranes
  193. Lentiviral-Mediated Gene Transfer to Model Triplet Repeat Disorders