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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

Detailed Technology Description: 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

Countries: Not Available

Application No.: None

*Abstract: 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

Country/Region: USA

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