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AssigneeEncoded Therapeutics, Inc.
InventorsKartik RAMAMOORTHI, Stephanie TAGLIATELA, Anne TANENHAUS, Andrew YOUNG, Szu-Ying CHEN, Chi ZHANG, Stephanie MARTIN, David OBERKOFLER, Victoria WONG, Jianmin LI
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Provided herein are nucleic acid molecules and vectors comprising variant copper transporting ATPase 2 (ATP7B) nucleic acid sequences. Such sequences have been optimized for expression in mammalian cells, liver cells, and/or from an adeno associated viral vector (AAV), including truncated and/or codon optimized variants. Also provided are viral vectors comprising such ATP7B variant nucleic acid sequences, and methods of use thereof for treating disorders associated with an ATP7B deficiency, such as Wilsons disease.
A nucleic acid molecule comprising a nucleotide sequence having (i) at least 80% sequence identity to SEQ ID NO:
A nucleic acid molecule comprising:
A nucleic acid molecule comprising:
A nucleic acid molecule comprising:
A nucleic acid molecule comprising:
Dependencies are inferred from the claim text.