Characterization of 12 AAV Vectors for Intravascular Delivery to Target CNS and Detarget Non-CNS Tissues by MIRNA Regulation: Implications in Treatment of Canavan Disease
- 详细技术说明
- New effective and safe gene therapy approach that allows gene delivery across the blood brain barrier (BBB) upon intravascular administration. Designed to minimize off-target effects by incorporation
- *Abstract
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New effective and safe gene therapy approach that allows gene delivery across the blood brain barrier (BBB) upon intravascular administration. Designed to minimize off-target effects by incorporation of non-CNS-tissue specific miRNA binding site into the transgene expression cassette.
- *IP Issue Date
- None
- *IP Type
- Other Patent
- 国家
- Not Available
- 申请号码
- None
- 国家/地区
- 美国
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