Compositions and methods for selective gene regulation
AssigneeEncoded Therapeutics, Inc.
InventorsStephanie TAGLIATELA, Anne TANENHAUS, Kartik RAMAMOORTHI, Andrew YOUNG, David OBERKOFLER
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Abstract
Provided herein are engineered transcription factors for selective upregulation of SCN1a and uses thereof for treating diseases and disorders, such as, Dravet syndrome. Also provided are microRNA binding sites and uses thereof for selective expression in parvalbumin neurons.
Claims60
1-91. (canceled)
A method of increasing SCN1A expression in a cell comprising contacting the cell with a polynucleotide comprising an expression cassette, wherein the expression cassette comprises a regulatory element operably linked to a nucleic acid sequence encoding an engineered transcription factor, wherein the engineered transcription factor comprises a zinc finger DNA binding domain and a transcription activation domain, wherein the zinc finger DNA binding domain comprises an amino acid sequence comprising SEQ ID NO:
- 148.
93-116. (canceled)
A method of reducing seizures in a mammal in need thereof comprising administering to the mammal an effective amount of a polynucleotide comprising an expression cassette, wherein the expression cassette comprises a regulatory element operably linked to a nucleic acid sequence encoding an engineered transcription factor, wherein the engineered transcription factor comprises a zinc finger DNA binding domain and a transcription activation domain, wherein the zinc finger DNA binding domain comprises an amino acid sequence comprising SEQ ID NO:148.
A method of treating Dravet syndrome in a subject in need thereof comprising administering to the subject an effective amount of a polynucleotide comprising an expression cassette, wherein the expression cassette comprises a regulatory element operably linked to a nucleic acid sequence encoding an engineered transcription factor, wherein the engineered transcription factor comprises a zinc finger DNA binding domain and a transcription activation domain, wherein the zinc finger DNA binding domain comprises an amino acid sequence comprising SEQ ID NO:148.
Dependencies are inferred from the claim text.