| scholarly article | Q13442814 |
| P50 | author | James Wilson | Q16947166 |
| P2093 | author name string | Ronald G Crystal | |
| Daniel A Peterson | |||
| Neil R Hackett | |||
| Dolan Sondhi | |||
| Jamie Stratton | |||
| Michael Baad | |||
| Kelly M Travis | |||
| P2860 | cites work | Classical late infantile neuronal ceroid lipofuscinosis fibroblasts are deficient in lysosomal tripeptidyl peptidase I | Q22001448 |
| Association of mutations in a lysosomal protein with classical late-infantile neuronal ceroid lipofuscinosis | Q24311617 | ||
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration | Q28591640 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Efficient neuronal gene transfer with AAV8 leads to neurotoxic levels of tau or green fluorescent proteins | Q34330389 | ||
| Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors | Q34938807 | ||
| Immunological aspects of recombinant adeno-associated virus delivery to the mammalian brain | Q35000819 | ||
| The neuronal ceroid-lipofuscinoses | Q35045618 | ||
| Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. | Q37181003 | ||
| Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain | Q40329324 | ||
| Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin | Q40488137 | ||
| Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain | Q42280576 | ||
| Central neuronal loss and behavioral impairment in mice lacking neurotrophin receptor p75. | Q42466426 | ||
| Distribution of tripeptidyl peptidase I in human tissues under normal and pathological conditions | Q43540060 | ||
| Convection-enhanced delivery of AAV-2 combined with heparin increases TK gene transfer in the rat brain. | Q43660897 | ||
| Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain | Q44686125 | ||
| Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis | Q44933213 | ||
| Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis. | Q45026873 | ||
| Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain | Q45420843 | ||
| High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses | Q45422901 | ||
| Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum. | Q45552423 | ||
| Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. | Q45738002 | ||
| Characterization of intrastriatal recombinant adeno-associated virus-mediated gene transfer of human tyrosine hydroxylase and human GTP-cyclohydrolase I in a rat model of Parkinson's disease. | Q45756120 | ||
| Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector | Q45769447 | ||
| Viral-mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP-I to the mouse central nervous system | Q45857721 | ||
| Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. | Q45864505 | ||
| Use of quantitative TaqMan real-time PCR to track the time-dependent distribution of gene transfer vectors in vivo | Q45873446 | ||
| Prevention of neuropathology in the mouse model of Hurler syndrome | Q45876472 | ||
| AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2. | Q45884747 | ||
| AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL. | Q45885520 | ||
| CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis. | Q45889316 | ||
| In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector | Q45889326 | ||
| Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates | Q45889555 | ||
| AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease | Q46455025 | ||
| Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. | Q48526007 | ||
| Long-term and significant correction of brain lesions in adult mucopolysaccharidosis type VII mice using recombinant AAV vectors. | Q51975070 | ||
| Targeted retrograde gene delivery for neuronal protection | Q77474905 | ||
| P433 | issue | 3 | |
| P921 | main subject | vector-borne disease | Q2083837 |
| Jansky–Bielschowsky disease | Q4354940 | ||
| Macaca | Q177601 | ||
| P1104 | number of pages | 11 | |
| P304 | page(s) | 481-491 | |
| P577 | publication date | 2006-12-19 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector | |
| P478 | volume | 15 |
| Q42370349 | A canine model for neuronal ceroid lipofuscinosis highlights the promise of gene therapy for lysosomal storage diseases |
| Q38075068 | A next step in adeno-associated virus-mediated gene therapy for neurological diseases: regulation and targeting. |
| Q44453459 | A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease |
| Q33659389 | AAV's anatomy: roadmap for optimizing vectors for translational success |
| Q34330377 | AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method |
| Q41655580 | AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy. |
| Q48510056 | AAVrh.10-mediated APOE2 CNS Gene Therapy for APOE4-associated Alzheimer's Disease |
| Q92069614 | Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation |
| Q38703339 | Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration |
| Q47102617 | Adeno-associated virus serotype rh10 is a useful gene transfer vector for sensory nerves that innervate bone in immunodeficient mice. |
| Q34063628 | Adeno-associated virus serotypes 1, 8, and 9 share conserved mechanisms for anterograde and retrograde axonal transport. |
| Q37434391 | Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain. |
| Q27026177 | Adeno-associated virus-mediated cancer gene therapy: current status |
| Q34770058 | Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection |
| Q36155208 | Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma |
| Q21129328 | Biology of adeno-associated viral vectors in the central nervous system |
| Q30394711 | Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease |
| Q36615488 | CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington's disease. |
| Q40495360 | Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism |
| Q37717896 | Chronic Enzyme Replacement to the Brain of a Late Infantile Neuronal Ceroid Lipofuscinosis Mouse Has Differential Effects on Phenotypes of Disease |
| Q35899850 | Clinical applications involving CNS gene transfer |
| Q42198683 | Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates |
| Q35853990 | Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids. |
| Q36147497 | Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice. |
| Q27653009 | Crystal Structure and Autoactivation Pathway of the Precursor Form of Human Tripeptidyl-peptidase 1, the Enzyme Deficient in Late Infantile Ceroid Lipofuscinosis |
| Q35158684 | Dipeptidyl-peptidase I does not functionally compensate for the loss of tripeptidyl-peptidase I in the neurodegenerative disease late-infantile neuronal ceroid lipofuscinosis |
| Q35559945 | Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I. |
| Q33484220 | Directed evolution of adeno-associated virus for glioma cell transduction |
| Q37099056 | Evaluating Permeability Surface-Area Product as a Measure of Blood-Brain Barrier Permeability in a Murine Model. |
| Q27318087 | Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII. |
| Q36401067 | Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease |
| Q39851951 | Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations. |
| Q39339697 | Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I. |
| Q30357745 | Gene therapy for metachromatic leukodystrophy. |
| Q38200599 | Gene therapy for the neurological manifestations in lysosomal storage disorders |
| Q22241423 | Gene therapy in Parkinson's disease: rationale and current status |
| Q34055904 | Gene transfer to the CNS using recombinant adeno-associated virus |
| Q34943946 | Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma |
| Q37649077 | Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway |
| Q35258046 | Human serum albumin-based nanoparticle-mediated in vitro gene delivery |
| Q34714906 | Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption |
| Q39415889 | Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats |
| Q58255075 | Intracranial Stem Cell-Based Transplantation: Reconsidering the Ethics of Phase 1 Clinical Trials in Light of Irreversible Interventions in the Brain |
| Q42246858 | Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy |
| Q45340805 | Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis |
| Q35285680 | Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis |
| Q35174910 | Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease. |
| Q30573520 | Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is trafficked in a highly motile Rab7-positive compartment |
| Q37358381 | Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis. |
| Q57039837 | Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease |
| Q36299764 | Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A |
| Q26753109 | Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis |
| Q36649689 | Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10 |
| Q43068346 | NCL in animal models |
| Q52598187 | Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses. |
| Q36909371 | Novel candidate disease for gene therapy: metachromatic leukodystrophy |
| Q36014074 | Novel therapies, high-risk pediatric research, and the prospect of benefit: learning from the ethical disagreements |
| Q30574131 | Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene. |
| Q41091625 | Persistent suppression of ocular neovascularization with intravitreal administration of AAVrh.10 coding for bevacizumab |
| Q42226636 | Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia |
| Q47548337 | Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis. |
| Q37563484 | Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors |
| Q92802302 | Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain |
| Q36772344 | Residual levels of tripeptidyl-peptidase I activity dramatically ameliorate disease in late-infantile neuronal ceroid lipofuscinosis |
| Q41988291 | Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis |
| Q35874710 | Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors. |
| Q35894587 | Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice |
| Q34365749 | Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome |
| Q59353538 | THE JEREMIAH METZGER LECTURE NOVEL THERAPEUTIC STRATEGIES OF ALLERGIC AND IMMUNOLOGIC DISORDERS |
| Q39996419 | Tau expression levels from various adeno-associated virus vector serotypes produce graded neurodegenerative disease states |
| Q35876224 | The AAV vector toolkit: poised at the clinical crossroads |
| Q38004089 | The advent of AAV9 expands applications for brain and spinal cord gene delivery |
| Q36784566 | Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses. |
| Q98945206 | Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease |
| Q37866086 | Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges |
| Q64040442 | Therapeutic landscape for Batten disease: current treatments and future prospects |
| Q30541192 | Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy. |
| Q44039833 | Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease |
| Q42200227 | Transduction efficiency of neurons and glial cells by AAV-1, -5, -9, -rh10 and -hu11 serotypes in rat spinal cord following contusion injury |
| Q41643417 | Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3-Associated Retinal Degeneration |
| Q38029645 | Viral vectors and delivery strategies for CNS gene therapy |
| Q36960666 | Viral vectors for in vivo gene transfer in Parkinson's disease: properties and clinical grade production. |
| Q35226105 | When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials |
| Q36951265 | Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector |
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