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Preclinical Gene Therapy in a Large Animal Model of DMD

Preclinical Gene Therapy in a Large Animal Model of DMD
DMD 大型动物模型的临床前基因治疗
批准号：
7509210
负责人：
Xiao Xiao
金额：
$13.84万
依托单位：
UNIVERSITY OF PITTSBURGH AT PITTSBURGH
依托单位国家：
美国
项目类别：
财政年份：
2007
资助国家：
美国
项目状态：
已结题
起止时间：
2007-06-01 至 2009-05-31
来源：
https://reporter.nih.gov/project-details/7509210
关键词：
Animal ModelAnimalsBiochemicalCanis familiarisCell membraneChronicClinicalCollaborationsComplementary DNAConditionDependovirusDuchenne muscular dystrophyDystrophinEnvironmentExhibitsFactor IXGene DeliveryGene ExpressionGene TransferGenesGeneticGoalsHeartHistopathologyHumanImmuneImmune responseInflammationInjection of therapeutic agentIntramuscularIntraperitoneal InjectionsInvasiveLaboratoriesLacZ GenesLegLimb structureMediatingMethodsMissouriModelingMusMuscleMuscular DystrophiesMyopathyNon-Viral VectorNormal CellOutcomePathologyPatientsPerfusionProteinsPurposeReporterReporter GenesRouteSymptomsTestingTherapeutic EffectTransducersUniversitiesViraladeno-associated viral vectorgene therapyimmunogenicimprovedin vivomalemdx mousemini-dystrophinnovelpre-clinicalresponsesizevector

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Gene delivery to muscle and nerve for laminin-alpha2-deficient MD (MDC1A)
Gene delivery for fukutin-related protein deficiencies.
Gene delivery for fukutin-related protein deficiencies.
Gene delivery to muscle and nerve for laminin-alpha2-deficient MD (MDC1A)
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