All Stories

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