Tissue selective transgene expression
AssigneeEncoded Therapeutics, Inc.
InventorsStephanie Tagliatela, Andrew Young, Szu-Ying Chen, Kartik Ramamoorthi, David Oberkofler
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Abstract
Provided herein are compositions and methods for selective expression of a transgene. Compositions and methods for selective expression of a transgene comprise one or more human regulatory elements, which, when operably linked to a transgene, can facilitate selective expression of a transgene (for example, cell-type selective expression) in a target cell as compared to at least one or more non-target cells.
Claims10
A viral particle comprising a viral vector, wherein the viral vector comprises an expression cassette comprising a regulatory element operably linked to a heterologous transgene, wherein the regulatory element comprises a nucleic acid sequence with at least 95% identity to SEQ ID NO:
- 1 or 8, and wherein the expression cassette selectively increases expression of the heterologous transgene in parvalbumin (PV) neurons compared to non-PV cells.
Dependencies are inferred from the claim text.