Gene therapies for neurodegenerative disease
AssigneePrevail Therapeutics, Inc.
InventorsAsa Abeliovich, Laura Heckman, Herve Rhinn
Life of this patent
Estimating the term…
Abstract
The disclosure relates, in some aspects, to compositions and methods for treatment of neurodegenerative disease, for example Alzheimer's disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding an APOE protein isoform or a portion thereof, an inhibitory nucleic acid targeting an APOE gene or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of treating Alzheimer's disease by administering an expression construct to a subject in need thereof.
Claims25
An isolated nucleic acid comprising an expression construct encoding an inhibitory nucleic acid that inhibits expression or activity of APOE4 and a transgene that expresses APOE2.
An isolated nucleic acid comprising an expression construct encoding an APOE2 protein, wherein the isolated nucleic acid comprises the sequence set forth in SEQ ID NO:
- 4.
An isolated nucleic acid comprising an expression construct encoding an inhibitory nucleic acid that inhibits expression or activity of APOE4.
Dependencies are inferred from the claim text.