US2021040504A1PendingUtilityA1
Method of increasing the function of an aav vector
Est. expiryApr 7, 2025(expired)· nominal 20-yr term from priority
C12N 2750/14143A61K 48/005C12N 2750/14122A61P 43/00A61P 37/00C12N 15/86C12N 2750/14142A61K 48/0091A61K 48/00C07K 14/005A61P 31/12C12N 2750/14152C12N 7/00
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Claims
Abstract
A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and/or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.
Claims
exact text as granted — not AI-modified1 . An adeno-associated virus (AAV) comprising an AAV capsid and a minigene having AAV inverted terminal repeats and a heterologous gene operably linked to regulatory sequences which direct expression of the heterologous gene in a host cell, wherein the AAV capsid comprises AAV vp1 proteins, AAV vp2 proteins, and AAV vp3 proteins, wherein the AAV vp1 proteins have the sequence of amino acids 1 to 736 of SEQ ID NO: 29 with a K531E modification (AAV6.1).
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