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.
Claims71
A polynucleotide comprising a nucleic acid sequence encoding an engineered DNA binding protein comprising a zinc finger DNA binding domain, wherein the zinc finger DNA binding domain comprises an amino acid sequence comprising SEQ ID NO:
- 148.
A non-naturally occurring transcription factor comprising a DNA binding domain and a transcription activation domain, wherein the DNA binding domain comprises SEQ ID NO:148.
Dependencies are inferred from the claim text.