大脑转化 AAV 传递平台-猫眼课题宝

大脑转化 AAV 传递平台

批准号：

8696895

负责人：

Krystof S Bankiewicz

金额：

$55.35万

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/8696895

关键词：

Address Axonal Transport Behavior Brain Brain Stem Brain region Cannulas Cellular Tropism Clinic Clinical Clinical Trials Communities Corpus striatum structure Development Disease Employee Strikes FDA approved Gene Delivery Gene Transduction Agent Gray unit of radiation dose Human Image Immune response Infusion procedures Investments Island Magnetic Resonance Imaging Monitor Movement Multiple Sclerosis Nature Neurologic Neurons Neurosurgeon Oligodendroglia Patients Performance Primates Procedures Property Protocols documentation Relative (related person)Research Personnel Resources Safety Scientist Serotyping Specificity Structure System Techniques Technology Thalamic structure Therapeutic Therapeutic Agents Tracer Viral Vector adeno-associated viral vector anterograde transport base clinically relevant design empowered gene therapy gray matter improved in vivo millimeter nervous system disorder neurosurgery nonhuman primate programs putamen response transgene expression vector white matter

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中文摘要

翻译

描述（由申请人提供）：为了将神经基因治疗应用推进到临床，迫切需要提高我们对基因递送载体的了解，特别强调直接大脑递送后的分布和安全性。虽然AAV2目前在临床开发中占主导地位，但替代的AAV血清型可能更适合于某些应用。最近改变游戏规则的图像引导递送技术的发展提供了在直接脑递送后准确比较不同载体的机会。因此，我们的目的是研究5种AAV血清型（AAV 1、2、5、8和9）在限制性递送至非人灵长类动物（NHP）脑中的灰质或白色物质结构后的分布和免疫应答。这种对AAV载体的深入评估将为研究者提供科学的合理性，以选择特定的神经学应用的特定载体。

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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