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US 2026/0250646 A1Published application
Compositions and methods for epigenetic regulation of pcsk9 expression
AssigneesChroma Medicine, Inc., nChroma Bio, Inc.
InventorsNoorussahar Abubucker, Ari Friedland, Morgan Maeder, Vic Myer, Frederic Tremblay, Mary Shirley
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Abstract
This application relates to compositions and methods comprising epigenetic editors for epigenetic modification of PCSK9, as well as nucleic acids and vectors encoding the same. Also disclosed are cells epigenetically modified by the epigenetic editors.
Claims48
A system for repressing transcription of a human PCSK9 gene in a human cell, optionally a human hepatocyte, comprising
- a) one or more fusion proteins that collectively comprise
- a DNA methyltransferase (DNMT) domain and/or a domain that recruits a DNMT, optionally wherein the DNMT domain and/or the recruiter domain comprise a DNMT3A domain and/or a DNMT3L domain, and optionally wherein the recruited DNMT is DNMT3A, and
- a transcriptional repressor domain,
- each domain being linked to a DNA-binding domain that binds to a target region in the human PCSK9 gene;
- or
- b) one or more nucleic acid molecules encoding the one or more fusion proteins.
3 more elements
Dependencies are inferred from the claim text.