Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. - Wikidata - wikimedia - TriplyDB

Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.

Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.

http://www.wikidata.org/entity/Q36507871

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Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis Rett Syndrome: Crossing the Threshold to Clinical Translation MECP2 disorders: from the clinic to mice and back Therapeutic approaches for shankopathies Rett Syndrome: Reaching for Clinical Trials FMRP Expression Levels in Mouse Central Nervous System Neurons Determine Behavioral Phenotype.Treatment of congenital neurotransmitter deficiencies by intracerebral ventricular injection of an adeno-associated virus serotype 9 vector.Mice with an isoform-ablating Mecp2 exon 1 mutation recapitulate the neurologic deficits of Rett syndrome.Twenty-four hour quantitative-EEG and in-vivo glutamate biosensor detects activity and circadian rhythm dependent biomarkers of pathogenesis in Mecp2 null mice.Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery.Intravascular AAV9 Administration for Delivering RNA Silencing Constructs to the CNS and Periphery.Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome Reduced phenotypic severity following adeno-associated virus-mediated Fmr1 gene delivery in fragile X mice.Gait analysis in a Mecp2 knockout mouse model of Rett syndrome reveals early-onset and progressive motor deficits Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain.Biomechanical properties of bone in a mouse model of Rett syndrome.Gene therapy for the nervous system: challenges and new strategies.Clinical applications involving CNS gene transfer.Methods for gene transfer to the central nervous system.Recent molecular approaches to understanding astrocyte function in vivo.The effect of brief neonatal cryoanesthesia on physical development and adult cognitive function in mice.Neurobiologically-based treatments in Rett syndrome: opportunities and challenges.Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington's disease mice.Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.AAV9-based gene therapy partially ameliorates the clinical phenotype of a mouse model of Leigh syndrome.Genetic and Pharmacological Reversibility of Phenotypes in Mouse Models of Autism Spectrum Disorder.The Role of Noncoding RNAs in Neurodevelopmental Disorders: The Case of Rett Syndrome.B-type natriuretic peptide overexpression ameliorates hepatorenal fibrocystic disease in a rat model of polycystic kidney disease.Lessons learned from studying syndromic autism spectrum disorders.Transduction of the central nervous system after intracerebroventricular injection of adeno-associated viral vectors in neonatal and juvenile mice Radically truncated MeCP2 rescues Rett syndrome-like neurological defects.Exclusive expression of MeCP2 in the nervous system distinguishes between brain and peripheral Rett syndrome-like phenotypes.Site-directed RNA repair of endogenous Mecp2 RNA in neurons.Rett syndrome from bench to bedside: recent advances.Autism spectrum disorder: prospects for treatment using gene therapy.Gene therapy for Rett syndrome: prospects and challenges

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Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice.

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Jie V Deng

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Rosemary C Spike

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P2860

Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus

Expression of MeCP2 in postmitotic neurons rescues Rett syndrome in mice

Rett syndrome: revised diagnostic criteria and nomenclature

Adeno-associated virus: from defective virus to effective vector

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

A mouse Mecp2-null mutation causes neurological symptoms that mimic Rett syndrome

Insight into Rett syndrome: MeCP2 levels display tissue- and cell-specific differences and correlate with neuronal maturation

Mecp2 deficiency disrupts norepinephrine and respiratory systems in mice

Mecp2 deficiency is associated with learning and cognitive deficits and altered gene activity in the hippocampal region of mice

Developmental expression of methyl-CpG binding protein 2 is dynamically regulated in the rodent brain

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18-30

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10.1038/MT.2012.200

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1

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231210733

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3536818

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