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AssigneesSangamo Therapeutics, Inc., Shire Human Genetic Therapies, Inc.
InventorsGalen Carey, Matthew Chiocco, Vivian Choi, Brian Felice, Steven Froelich, Debra Klatte, Jeffrey Miller, David Paschon, Edward Rebar, Bryan Zeitler, Lei Zhang, H. Steve Zhang
Estimating the term…
Disclosed herein are HTT repressors and methods and compositions for use of these HTT repressors. Disclosed herein are methods and compositions for diagnosing, preventing and/or treating Huntington's Disease. In particular, provided herein are methods and compositions for modifying (e.g., modulating expression of) an HD HTT allele so as to prevent or treat Huntington Disease, including mHTT repressors (that repress mHTT transcripts and thus also repress mHTT protein expression).
A zinc finger protein transcription factor (ZFP-TF) comprising a zinc finger protein (ZFP) designated 45294 or 45723 or comprising the amino acid sequence of a ZFP-TF as shown in Table 3.
Dependencies are inferred from the claim text.