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Translational AAV Delivery Platform to the Brain

Translational AAV Delivery Platform to the Brain
大脑转化 AAV 传递平台
批准号：
8303199
负责人：
Krystof S Bankiewicz
金额：
$60.12万
依托单位：
UNIVERSITY OF CALIFORNIA, SAN FRANCISCO
依托单位国家：
美国
项目类别：
财政年份：
2011
资助国家：
美国
项目状态：
已结题
起止时间：
2011-08-01 至 2016-07-31
来源：
https://reporter.nih.gov/project-details/8303199
关键词：
AddressAxonal TransportBehaviorBrainBrain StemBrain regionCannulasCellular TropismClinicClinicalClinical TrialsCommunitiesCorpus striatum structureDevelopmentDiseaseEmployee StrikesFDA approvedGene DeliveryGene Transduction AgentGray unit of radiation doseHumanImageImmune responseInfusion proceduresInvestmentsIslandMagnetic Resonance ImagingMonitorMovementMultiple SclerosisNatureNeurologicNeuronsNeurosurgeonOligodendrogliaPatientsPerformancePrimatesProceduresPropertyProtocols documentationRelative (related person)Research PersonnelResourcesSafetyScientistSerotypingSpecificityStructureSystemTechniquesTechnologyThalamic structureTherapeuticTherapeutic AgentsTracerViral Vectoradeno-associated viral vectoranterograde transportbaseclinically relevantdesignempoweredgene therapygray matterimprovedin vivomillimeternervous system disorderneurosurgerynonhuman primateprogramsputamenresponsetransgene expressionvectorwhite matter

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英文摘要
DESCRIPTION (provided by applicant): To advance neurological gene therapy applications into the clinic there is an urgent need to improve our understanding of gene delivery vectors, with particular emphasis on distribution and safety after direct brain delivery. While AAV2 currently dominates clinical development, alternative AAV serotypes may be better suited for some applications. The recent game-changing development of image-guided delivery techniques provides opportunity to accurately compare different vectors after direct brain delivery. Accordingly we aim to investigate the distribution and immune responses of 5 AAV serotypes (AAV1, 2, 5, 8 and 9) after confined delivery to grey or white matter structures in the non-human primate (NHP) brain. This in-depth assessment of AAV vectors will provide investigators with scientific rational for selecting a specific vector for a particular neurological application.
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