| scholarly article | Q13442814 |
| review article | Q7318358 |
| P2093 | author name string | Raymond T Bartus | |
| Marc S Weinberg | |||
| R. Jude Samulski | |||
| P2860 | cites work | Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus | Q24523073 |
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| Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012 | Q28708928 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Association of cerebrospinal fluid β-amyloid 1-42, T-tau, P-tau181, and α-synuclein levels with clinical features of drug-naive patients with early Parkinson disease | Q33666297 | ||
| Comparative transduction efficiency of AAV vector serotypes 1-6 in the substantia nigra and striatum of the primate brain | Q33731226 | ||
| Safety and tolerability of putaminal AADC gene therapy for Parkinson disease | Q33731687 | ||
| Safety evaluation of AAV2-GDNF gene transfer into the dopaminergic nigrostriatal pathway in aged and parkinsonian rhesus monkeys | Q33819946 | ||
| A pathologic cascade leading to synaptic dysfunction in alpha-synuclein-induced neurodegeneration. | Q33982546 | ||
| Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. | Q34039452 | ||
| alpha -Synucleinopathy and selective dopaminergic neuron loss in a rat lentiviral-based model of Parkinson's disease. | Q34076094 | ||
| Subthalamic GAD gene therapy in a Parkinson's disease rat model | Q34154186 | ||
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 | ||
| Adeno-associated virus vectorology, manufacturing, and clinical applications | Q34172314 | ||
| A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease | Q34209755 | ||
| AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method | Q34330377 | ||
| Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. | Q34397099 | ||
| α-Synuclein-induced down-regulation of Nurr1 disrupts GDNF signaling in nigral dopamine neurons | Q34503671 | ||
| Gene therapy for Parkinson's disease | Q83053649 | ||
| The long march of antisense | Q84237894 | ||
| Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo | Q45709105 | ||
| Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. | Q45859568 | ||
| Subthalamic glutamic acid decarboxylase gene therapy: changes in motor function and cortical metabolism | Q45859700 | ||
| Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy | Q45884700 | ||
| Intracerebroventricular infusion of nerve growth factor in three patients with Alzheimer's disease | Q47695139 | ||
| Intraventricular infusion of nerve growth factor as the cause of sympathetic fiber sprouting in sensory ganglia | Q48122644 | ||
| Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys | Q48197305 | ||
| Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys | Q48325968 | ||
| Enhanced neurotrophic distribution, cell signaling and neuroprotection following substantia nigral versus striatal delivery of AAV2-NRTN (CERE-120). | Q51029408 | ||
| Treatment of septic shock with human monoclonal antibody HA-1A. A randomized, double-blind, placebo-controlled trial. CHESS Trial Study Group. | Q51132705 | ||
| Gene therapy for Parkinson's disease | Q58913419 | ||
| Efficacy of minocycline in patients with amyotrophic lateral sclerosis: a phase III randomised trial. | Q34584573 | ||
| Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial | Q34640765 | ||
| The scientific and clinical basis for the treatment of Parkinson disease (2009). | Q34983175 | ||
| Adenovirus-associated virus vector-mediated gene transfer in hemophilia B | Q35691659 | ||
| Challenges in Parkinson's disease: restoration of the nigrostriatal dopamine system is not enough | Q35751844 | ||
| Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease | Q35932749 | ||
| The birth pangs of monoclonal antibody therapeutics: the failure and legacy of Centoxin | Q35969881 | ||
| Nonmotor symptoms in Parkinson's disease in 2012: relevant clinical aspects | Q36135045 | ||
| Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients | Q37027502 | ||
| Disease duration and the integrity of the nigrostriatal system in Parkinson's disease | Q37043102 | ||
| An inducible system for highly efficient production of recombinant adeno-associated virus (rAAV) vectors in insect Sf9 cells | Q37125170 | ||
| Treatment of Parkinson's disease with trophic factors | Q37131259 | ||
| Dynamic changes in presynaptic and axonal transport proteins combined with striatal neuroinflammation precede dopaminergic neuronal loss in a rat model of AAV alpha-synucleinopathy. | Q37221349 | ||
| A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys | Q37300994 | ||
| Real-time MR imaging of adeno-associated viral vector delivery to the primate brain | Q37313954 | ||
| Focal striatal dopamine may potentiate dyskinesias in parkinsonian monkeys | Q37398168 | ||
| Repairing the parkinsonian brain with neurotrophic factors | Q37818693 | ||
| Adeno-associated virus (AAV) gene therapy for neurological disease | Q37998750 | ||
| Translating the therapeutic potential of neurotrophic factors to clinical 'proof of concept': a personal saga achieving a career-long quest | Q38004721 | ||
| Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating "clinical proof-of-concept" for AAV-neurturin (CERE-120) in Parkinson's disease | Q38038438 | ||
| GDNF fails to exert neuroprotection in a rat α-synuclein model of Parkinson's disease | Q39516461 | ||
| Properly scaled and targeted AAV2-NRTN (neurturin) to the substantia nigra is safe, effective and causes no weight loss: support for nigral targeting in Parkinson's disease | Q39518245 | ||
| Bioactivity of AAV2-neurturin gene therapy (CERE-120): differences between Parkinson's disease and nonhuman primate brains. | Q39782948 | ||
| Expression, bioactivity, and safety 1 year after adeno-associated viral vector type 2-mediated delivery of neurturin to the monkey nigrostriatal system support cere-120 for Parkinson's disease | Q39863620 | ||
| Six-month continuous intraputamenal infusion toxicity study of recombinant methionyl human glial cell line-derived neurotrophic factor (r-metHuGDNF in rhesus monkeys | Q40031959 | ||
| Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose | Q40039645 | ||
| Enhanced gene transfer efficiency in the murine striatum and an orthotopic glioblastoma tumor model, using AAV-7- and AAV-8-pseudotyped vectors | Q40238020 | ||
| Randomized controlled trial of intraputamenal glial cell line-derived neurotrophic factor infusion in Parkinson disease | Q40326219 | ||
| Lentiviral nigral delivery of GDNF does not prevent neurodegeneration in a genetic rat model of Parkinson's disease | Q40506120 | ||
| Randomized, double-blind trial of glial cell line-derived neurotrophic factor (GDNF) in PD. | Q40678042 | ||
| Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach | Q40870377 | ||
| Differential effects of glial cell line-derived neurotrophic factor and neurturin on developing and adult substantia nigra dopaminergic neurons | Q40945102 | ||
| Protection and regeneration of nigral dopaminergic neurons by neurturin or GDNF in a partial lesion model of Parkinson's disease after administration into the striatum or the lateral ventricle | Q40958690 | ||
| Gene transfer provides a practical means for safe, long-term, targeted delivery of biologically active neurotrophic factor proteins for neurodegenerative diseases | Q41195395 | ||
| Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver | Q41753510 | ||
| Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC. | Q42502571 | ||
| 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 | Q42650874 | ||
| Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia. | Q43109280 | ||
| Parkinson-like neurodegeneration induced by targeted overexpression of alpha-synuclein in the nigrostriatal system. | Q43940212 | ||
| Minocycline inhibits cytochrome c release and delays progression of amyotrophic lateral sclerosis in mice | Q43977943 | ||
| A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease | Q44571920 | ||
| Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum | Q44631221 | ||
| Gene therapy for aromatic L-amino acid decarboxylase deficiency | Q45215332 | ||
| Real-time imaging of convection-enhanced delivery of viruses and virus-sized particles | Q45399982 | ||
| P275 | copyright license | Creative Commons Attribution-NonCommercial-ShareAlike 3.0 Unported | Q15643954 |
| P6216 | copyright status | copyrighted | Q50423863 |
| P433 | issue | 3 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | nervous system | Q9404 |
| gene therapy | Q213901 | ||
| Parkinson's disease | Q11085 | ||
| biomedical investigative technique | Q66648976 | ||
| P5008 | on focus list of Wikimedia project | ScienceSource | Q55439927 |
| P304 | page(s) | 487-97 | |
| P577 | publication date | 2014-03-01 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Parkinson's disease gene therapy: success by design meets failure by efficacy | |
| P478 | volume | 22 |
| Q36744851 | A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses |
| Q34464281 | Beyond the hammer and the scalpel: selective circuit control for the epilepsies |
| Q40643280 | CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector |
| Q91891770 | Cell-based interferon gene therapy using proliferation-controllable, interferon-releasing mesenchymal stem cells |
| Q58611364 | Cellular and Molecular Aspects of Parkinson Treatment: Future Therapeutic Perspectives |
| Q41623081 | Chaperone-Based Therapies for Disease Modification in Parkinson's Disease. |
| Q55282219 | Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. |
| Q38744447 | Convection-Enhanced Delivery |
| Q33627771 | Current Experimental Studies of Gene Therapy in Parkinson's Disease |
| Q38652153 | Current disease modifying approaches to treat Parkinson's disease |
| Q58805419 | Current surgical treatments for Parkinson's disease and potential therapeutic targets |
| Q40264729 | Deficiency in Neuronal TGF-β Signaling Leads to Nigrostriatal Degeneration and Activation of TGF-β Signaling Protects against MPTP Neurotoxicity in Mice |
| Q39610770 | Delivering therapy to target: improving the odds for successful drug development. |
| Q38761724 | Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery |
| Q99711416 | Effectiveness of Acupuncture in the Treatment of Parkinson's Disease: An Overview of Systematic Reviews |
| Q52721898 | GDNF-expressing macrophages mitigate loss of dopamine neurons and improve Parkinsonian symptoms in MitoPark mice. |
| Q45873346 | Gene delivery by a cationic and thermosensitive nanogel promoted established tumor growth inhibition |
| Q41681737 | Gene delivery of neurturin to putamen and substantia nigra in Parkinson disease: A double-blind, randomized, controlled trial |
| Q43154354 | Gene therapy for Parkinson's disease: a decade of progress supported by posthumous contributions from volunteer subjects |
| Q35340196 | Gene therapy for the nervous system: challenges and new strategies |
| Q41020507 | Gene therapy in Parkinson's disease: targeting the endplasmic reticulum proteostasis network |
| Q38957332 | Gene therapy targeting mitochondrial pathway in Parkinson's disease |
| Q37742852 | Glial Cell Line-Derived Neurotrophic Factor Gene Delivery in Parkinson's Disease: A Delicate Balance between Neuroprotection, Trophic Effects, and Unwanted Compensatory Mechanisms. |
| Q57829929 | Glycomic and Proteomic Changes in Aging Brain Nigrostriatal Pathway |
| Q37277317 | Glymphatic fluid transport controls paravascular clearance of AAV vectors from the brain |
| Q38815321 | Increasing uncertainty in CNS clinical trials: the role of placebo, nocebo, and Hawthorne effects |
| Q90016860 | Intracerebral Delivery in Complex 3D Arrays: The Intracerebral Microinjection Instrument |
| Q60111586 | Ion-Catalyzed Reactive Oxygen Species in Sporadic Models of Parkinson’s Disease |
| Q38768061 | Iron and dopamine: a toxic couple. |
| Q26752578 | Lipopolyplex for Therapeutic Gene Delivery and Its Application for the Treatment of Parkinson's Disease |
| Q91895425 | Magnetic resonance imaging-guided phase 1 trial of putaminal AADC gene therapy for Parkinson's disease |
| Q26773485 | Merging DBS with viral vector or stem cell implantation: "hybrid" stereotactic surgery as an evolution in the surgical treatment of Parkinson's disease |
| Q38622674 | Microcircuits in Epilepsy: Heterogeneity and Hub Cells in Network Synchronization |
| Q28087559 | Neuroelectronics and Biooptics: Closed-Loop Technologies in Neurological Disorders |
| Q35878528 | Neurotensin-polyplex-mediated brain-derived neurotrophic factor gene delivery into nigral dopamine neurons prevents nigrostriatal degeneration in a rat model of early Parkinson's disease |
| Q47162932 | Neurturin overexpression in dopaminergic neurons induces presynaptic and postsynaptic structural changes in rats with chronic 6-hydroxydopamine lesion |
| Q48411251 | Nigral dopaminergic PAK4 prevents neurodegeneration in rat models of Parkinson's disease |
| Q92345091 | Non-toxic HSC Transplantation-Based Macrophage/Microglia-Mediated GDNF Delivery for Parkinson's Disease |
| Q38834011 | Optogenetic Approaches for Controlling Seizure Activity. |
| Q64904807 | Parkinson's Disease: Biomarkers, Treatment, and Risk Factors. |
| Q52589683 | Pluripotent stem cell-based therapy for Parkinson's disease: Current status and future prospects. |
| Q53820484 | Potential genetic modifiers of disease risk and age at onset in patients with frontotemporal lobar degeneration and GRN mutations: a genome-wide association study. |
| Q38949089 | Pre-existing immunity to adeno-associated virus (AAV)2 limits transgene expression following intracerebral AAV2-based gene delivery in a 6-hydroxydopamine model of Parkinson's disease. |
| Q38767711 | Promising therapeutic agents for the treatment of Parkinson's disease |
| Q36690552 | Promotion of mitochondrial biogenesis by necdin protects neurons against mitochondrial insults |
| Q38803772 | Prospect of ultrasound-mediated gene delivery in cardiovascular applications |
| Q30665945 | Raster image cross-correlation analysis for spatiotemporal visualization of intracellular degradation activities against exogenous DNAs |
| Q38520864 | Rational drug discovery design approaches for treating Parkinson's disease |
| Q40434024 | Recombinant Adeno-Associated Virus Serotype 6 (rAAV6) Potently and Preferentially Transduces Rat Astrocytes In vitro and In vivo. |
| Q40133309 | Recombinant elastin-based nanoparticles for targeted gene therapy |
| Q38369065 | Regenerative medicine for Parkinson's disease |
| Q42494301 | Regulating the expression of therapeutic transgenes by controlled intake of dietary essential amino acids |
| Q35180062 | Roles of Rheb(S16H) in substantia nigra pars compacta dopaminergic neurons in vivo |
| Q37481542 | Significant changes in endogenous retinal gene expression assessed 1 year after a single intraocular injection of AAV-CNTF or AAV-BDNF |
| Q89792742 | Strategies for the Treatment of Parkinson's Disease: Beyond Dopamine |
| Q34497784 | Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease |
| Q54113831 | Targeted therapies for Parkinson's disease: From genetics to the clinic. |
| Q34435001 | Targets for future clinical trials in Huntington's disease: what's in the pipeline? |
| Q90599504 | The Drainage of Interstitial Fluid in the Deep Brain is Controlled by the Integrity of Myelination |
| Q60950132 | The Future of Surgical Treatments for Parkinson's Disease |
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