Viral gene therapy as treatment for cholesterol storage disease or disorder

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United States of America Patent

PATENT NO 12201658
APP PUB NO 20210113635A1
SERIAL NO

17033166

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Abstract

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Provided herein are compositions and methods for the viral gene therapy (e.g., AAV-directed gene therapy) of cholesterol storage diseases or disorders, such as Niemann-Pick disease, Type C.

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Patent Owner(s)

  • THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY DEPARTMENT OF HEALTH AND HUMAN SERVICES

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Chandler, Randy Washington, US 6 12
Pavan, William J Derwood, US 9 43
Venditti, Charles P Kensington, US 23 33

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