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AssigneeEncoded Therapeutics, Inc.
InventorsAnne TANENHAUS, Ben ZHAO, Serena LIU, Puja DHANOTA, Raghavendra HOSUR, Steven TAN, John MCLAUGHLIN, Martin MOORHEAD, Sheila SEARS, Yosr BOUHLAL, Nathan MOERKE, Camille G. ARTUR, Mitchell LOPEZ, Tulasi Indrasinh SOLANKI, Greg LUCEY
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The present disclosure provides sequences that reduce expression of an operably linked transgene in dorsal root ganglion cells and/or liver cells. In some aspects, the sequences can be employed in gene therapy vectors to de-target expression of a therapeutic transgene in dorsal root ganglion and/or liver cells of a subject.
1-90. (canceled)
A nucleic acid cassette comprising a therapeutic transgene encoding an RNA transcript, wherein the RNA transcript comprises a sequence of (i) any of SEQ ID NO. 2 and SEQ ID NOs. 1, 3-10 and 43-48, (ii) a variant, functional fragment, or combination thereof, or (iii) a sequence at least 80% identical to (i) or (ii).
A method of decreasing dorsal root ganglion (DRG) expression of an RNA transcript and/or a therapeutic protein encoded thereby, the method comprising including a sequence of (i) any of SEQ ID NOs. 1-10 and 43-48, (ii) a variant, functional fragment, or combination thereof, or (iii) a sequence at least 80% identical to (i) or (ii) in the RNA transcript.
A nucleic acid cassette comprising a therapeutic transgene encoding an RNA transcript, wherein the RNA transcript comprises a sequence of (i) any SEQ ID NOS. 65, 110, and 112, (ii) a variant, functional fragment, or combination thereof, or (iii) a sequence at least 80% identical to (i) or (ii), wherein the sequence decreases expression of the mRNA in liver cells.
A method of decreasing liver expression of a therapeutic RNA transcript and/or a protein encoded thereby, the method comprising including a sequence of (i) any SEQ ID NOS. 65, 110, and 112, (ii) a variant, functional fragment, or combination thereof, or (iii) a sequence at least 80% identical to (i) or (ii) in the RNA transcript.
A nucleic acid cassette comprising a therapeutic transgene encoding an RNA transcript that comprises a first sequence that de-targets expression in dorsal root ganglion (DRG) cells and a second sequence that de-targets expression in liver cells, wherein:
A method of decreasing dorsal root ganglion (DRG) and liver expression of a therapeutic RNA transcript and/or a protein encoded thereby, the method comprising adding a first and second sequences to the therapeutic RNA transcript, wherein the first sequence de-targets expression in DRG cells and the second sequence de-targets expression in liver cells, wherein:
Dependencies are inferred from the claim text.