Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease. - Wikidata - awgldk - TriplyDB

Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.

Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.

http://www.wikidata.org/entity/Q36401067

about

Biology of adeno-associated viral vectors in the central nervous system Differential diagnosis of Mendelian and mitochondrial disorders in patients with suspected multiple sclerosis New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones Neuroimmune mechanisms in Krabbe's disease Gene therapy for metachromatic leukodystrophy.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.Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy.Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy On the role of natural killer cells in neurodegenerative diseases Experimental therapies in the murine model of globoid cell leukodystrophy.Effect of vitamin D3 intake on the onset of disease in a murine model of human Krabbe disease.Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia A microglial hypothesis of globoid cell leukodystrophy pathology.Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant Recent gene therapy advancements for neurological diseases.Cellular unfolded protein response against viruses used in gene therapy Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Molecular Mechanisms of Disease Pathogenesis Differ in Krabbe Disease Variants.Myelin repair by transplantation of myelin-forming cells in globoid cell leukodystrophy.Krabbe disease: One Hundred years from the bedside to the bench to the bedside.Biochemical, cell biological, pathological, and therapeutic aspects of Krabbe's disease.Treatment for Krabbe's disease: Finding the combination.Perspective on innovative therapies for globoid cell leukodystrophy.Immunological considerations for treating globoid cell leukodystrophy.HIV Tat Domain Improves Cross-correction of Human Galactocerebrosidase in a Gene- and Flanking Sequence-dependent Manner.Optimized AAV rh.10 Vectors That Partially Evade Neutralizing Antibodies during Hepatic Gene Transfer.Transduction efficiency of neurons and glial cells by AAV-1, -5, -9, -rh10 and -hu11 serotypes in rat spinal cord following contusion injury.Simulations suggest pharmacological methods for rescuing long-term potentiation.AAVrh10 gene therapy ameliorates central and peripheral nervous system disease in canine globoid cell leukodystrophy (Krabbe disease).AAVrh.10-mediated APOE2 CNS Gene Therapy for APOE4-associated Alzheimer's Disease.Suppression of galactocerebrosidase premature termination codon and rescue of galactocerebrosidase activity in twitcher cells.Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.

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Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.

http://www.wikidata.org/entity/Q36401067

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2012 nî lūn-bûn

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Extended normal life after AAV ...... mouse model of Krabbe disease.

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Molecular Therapy

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Extended normal life after AAV ...... mouse model of Krabbe disease.

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David A Wenger

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Han Zhi Rao

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Mark T Curtis

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Mohammad A Rafi

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Paola Luzi

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P2860

Protein measurement with the Folin phenol reagent

Molecular characterization of mutations that cause globoid cell leukodystrophy and pharmacological rescue using small molecule chemical chaperones

Anti-inflammatory therapy by ibudilast, a phosphodiesterase inhibitor, in demyelination of twitcher, a genetic demyelination model

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB).

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.

RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy.

Systemic administration of AAV8-α-galactosidase A induces humoral tolerance in nonhuman primates despite low hepatic expression

AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)

Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates

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2031-2042

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10.1038/MT.2012.153

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11

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20

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22850681

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