标题
Progress in gene therapy for neurological disorders
作者
关键词
-
出版物
Nature Reviews Neurology
Volume 9, Issue 5, Pages 277-291
出版商
Springer Nature
发表日期
2013-04-23
DOI
10.1038/nrneurol.2013.56
参考文献
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。- Gene Therapy in Large Animal Models of Human Genetic Diseases
- (2013) J. H. Wolfe ILAR JOURNAL
- Regeneration of the MPTP-Lesioned Dopaminergic System after Convection-Enhanced Delivery of AAV2-GDNF
- (2013) A. P. Kells et al. JOURNAL OF NEUROSCIENCE
- Identification of new epilepsy treatments: Issues in preclinical methodology
- (2012) Aristea S. Galanopoulou et al. EPILEPSIA
- Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound
- (2012) Emmanuel Thévenot et al. HUMAN GENE THERAPY
- A novel adenoviral vector labeled with superparamagnetic iron oxide nanoparticles for real-time tracking of viral delivery
- (2012) Jonathan Yun et al. JOURNAL OF CLINICAL NEUROSCIENCE
- Gene therapy rescues cilia defects and restores olfactory function in a mammalian ciliopathy model
- (2012) Jeremy C McIntyre et al. NATURE MEDICINE
- Restoration of Hearing in the VGLUT3 Knockout Mouse Using Virally Mediated Gene Therapy
- (2012) Omar Akil et al. NEURON
- Optogenetic and Potassium Channel Gene Therapy in a Rodent Model of Focal Neocortical Epilepsy
- (2012) R. C. Wykes et al. Science Translational Medicine
- Non-Viral Nucleic Acid Delivery: Key Challenges and Future Directions
- (2012) Mahmoud Elsabahy et al. Current Drug Delivery
- Gene therapy for pain: Results of a phase I clinical trial
- (2011) David J. Fink et al. ANNALS OF NEUROLOGY
- Gene Therapy and Targeted Toxins for Glioma
- (2011) Maria G. Castro et al. CURRENT GENE THERAPY
- Adenosine kinase as a target for therapeutic antisense strategies in epilepsy
- (2011) Panos Theofilas et al. EPILEPSIA
- Localized overexpression of FGF-2 and BDNF in hippocampus reduces mossy fiber sprouting and spontaneous seizures up to 4 weeks after pilocarpine-induced status epilepticus
- (2011) Beatrice Paradiso et al. EPILEPSIA
- Epidemiology and etiology of Parkinson’s disease: a review of the evidence
- (2011) Karin Wirdefeldt et al. EUROPEAN JOURNAL OF EPIDEMIOLOGY
- Vector-mediated release of GABA attenuates pain-related behaviors and reduces NaV1.7 in DRG neurons
- (2011) Munmun Chattopadhyay et al. EUROPEAN JOURNAL OF PAIN
- Prolonged regulatable expression of EPO from an HSV vector using the LAP2 promoter element
- (2011) Z Wu et al. GENE THERAPY
- Gene-based therapies for dominantly inherited retinopathies
- (2011) G J Farrar et al. GENE THERAPY
- Evaluation of the specificity and sensitivity of ferritin as an MRI reporter gene in the mouse brain using lentiviral and adeno-associated viral vectors
- (2011) G Vande Velde et al. GENE THERAPY
- Optogenetic therapy for retinitis pigmentosa
- (2011) V Busskamp et al. GENE THERAPY
- Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors
- (2011) Steven J. Gray et al. HUMAN GENE THERAPY
- The human visual cortex responds to gene therapy–mediated recovery of retinal function
- (2011) Manzar Ashtari et al. JOURNAL OF CLINICAL INVESTIGATION
- Phase IB Study of Gene-Mediated Cytotoxic Immunotherapy Adjuvant to Up-Front Surgery and Intensive Timing Radiation for Malignant Glioma
- (2011) E. Antonio Chiocca et al. JOURNAL OF CLINICAL ONCOLOGY
- AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial
- (2011) Peter A LeWitt et al. LANCET NEUROLOGY
- Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa
- (2011) Sophia Millington-Ward et al. MOLECULAR THERAPY
- Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates
- (2011) Steven J Gray et al. MOLECULAR THERAPY
- ATP7A Gene Addition to the Choroid Plexus Results in Long-term Rescue of the Lethal Copper Transport Defect in a Menkes Disease Mouse Model
- (2011) Anthony Donsante et al. MOLECULAR THERAPY
- Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease
- (2011) Jodi L McBride et al. MOLECULAR THERAPY
- Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer
- (2011) Nagesh Pulicherla et al. MOLECULAR THERAPY
- Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
- (2011) L. Jiang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Adeno-associated viral vector-induced overexpression of neuropeptide Y Y2 receptors in the hippocampus suppresses seizures
- (2010) David P. D. Woldbye et al. BRAIN
- Survival of Patients with Newly Diagnosed Glioblastoma Treated with Radiation and Temozolomide in Research Studies in the United States
- (2010) S. A. Grossman et al. CLINICAL CANCER RESEARCH
- Revised terminology and concepts for organization of seizures and epilepsies: Report of the ILAE Commission on Classification and Terminology, 2005-2009
- (2010) Anne T. Berg et al. EPILEPSIA
- Anticonvulsant effects and behavioural outcomes of rAAV serotype 1 vector-mediated neuropeptide Y overexpression in rat hippocampus
- (2010) F Noe et al. GENE THERAPY
- Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies
- (2010) Anneke I. den Hollander et al. JOURNAL OF CLINICAL INVESTIGATION
- Hippocampal FGF-2 and BDNF overexpression attenuates epileptogenesis-associated neuroinflammation and reduces spontaneous recurrent seizures
- (2010) Roberta Bovolenta et al. Journal of Neuroinflammation
- Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial
- (2010) William J Marks et al. LANCET NEUROLOGY
- Recent Advances in Lentiviral Vector Development and Applications
- (2010) Janka Mátrai et al. MOLECULAR THERAPY
- A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease
- (2010) Shin-ichi Muramatsu et al. MOLECULAR THERAPY
- HSV Delivery of a Ligand-regulated Endogenous Ion Channel Gene to Sensory Neurons Results in Pain Control Following Channel Activation
- (2010) James R Goss et al. MOLECULAR THERAPY
- Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes
- (2010) N Matthew Ellinwood et al. MOLECULAR THERAPY
- Systematic Comparison of Constitutive Promoters and the Doxycycline-Inducible Promoter
- (2010) Jane Yuxia Qin et al. PLoS One
- AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival
- (2010) Rena C. Baek et al. PLoS One
- Neuroprotective effect of herpes simplex virus-mediated gene transfer of erythropoietin in hyperglycemic dorsal root ganglion neurons
- (2009) M. Chattopadhyay et al. BRAIN
- A human trial of HSV-mediated gene transfer for the treatment of chronic pain
- (2009) D Wolfe et al. GENE THERAPY
- Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters
- (2009) T Husain et al. GENE THERAPY
- Progress and prospects: immune responses to viral vectors
- (2009) S Nayak et al. GENE THERAPY
- Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
- (2009) Sandra Duque et al. MOLECULAR THERAPY
- Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)
- (2009) Steven J Gray et al. MOLECULAR THERAPY
- Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
- (2009) Yong Hong Chen et al. NATURE MEDICINE
- Neuroprotective effects of brain-derived neurotrophic factor in rodent and primate models of Alzheimer's disease
- (2009) Alan H Nagahara et al. NATURE MEDICINE
- Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
- (2009) C. W. Christine et al. NEUROLOGY
- Localized delivery of fibroblast growth factor-2 and brain-derived neurotrophic factor reduces spontaneous seizures in an epilepsy model
- (2009) B. Paradiso et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
- (2009) N. Cartier et al. SCIENCE
- Dopamine Gene Therapy for Parkinson's Disease in a Nonhuman Primate Without Associated Dyskinesia
- (2009) B. Jarraya et al. Science Translational Medicine
- HMGB1 Mediates Endogenous TLR2 Activation and Brain Tumor Regression
- (2009) James F Curtin et al. PLOS MEDICINE
- Neuropeptide Y gene therapy decreases chronic spontaneous seizures in a rat model of temporal lobe epilepsy
- (2008) Francesco Noè et al. BRAIN
- Hippocampal NPY gene transfer attenuates seizures without affecting epilepsy-induced impairment of LTP
- (2008) Andreas T. Sørensen et al. EXPERIMENTAL NEUROLOGY
- GFAPpromoter elements required for region-specific and astrocyte-specific expression
- (2008) Youngjin Lee et al. GLIA
- Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA
- (2008) Stefan Worgall et al. HUMAN GENE THERAPY
- Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
- (2008) William W. Hauswirth et al. HUMAN GENE THERAPY
- Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial
- (2008) William J Marks et al. LANCET NEUROLOGY
- A Phase I Trial of Ad.hIFN-β Gene Therapy for Glioma
- (2008) E Antonio Chiocca et al. MOLECULAR THERAPY
- A Phase I Trial of Intratumoral Administration of Reovirus in Patients With Histologically Confirmed Recurrent Malignant Gliomas
- (2008) Peter Forsyth et al. MOLECULAR THERAPY
- Phase Ib Trial of Mutant Herpes Simplex Virus G207 Inoculated Pre-and Post-tumor Resection for Recurrent GBM
- (2008) James M Markert et al. MOLECULAR THERAPY
- Functional auditory hair cells produced in the mammalian cochlea by in utero gene transfer
- (2008) Samuel P. Gubbels et al. NATURE
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
- (2008) Kevin D Foust et al. NATURE BIOTECHNOLOGY
- Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington's disease
- (2008) Shilpa Ramaswamy et al. NEUROBIOLOGY OF DISEASE
- Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS
- (2008) Colin K. Franz et al. NEUROBIOLOGY OF DISEASE
- Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
- (2008) Albert M. Maguire et al. NEW ENGLAND JOURNAL OF MEDICINE
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
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