Methods and compositions for treating Huntington's Disease
AssigneeSangamo Therapeutics, Inc.
InventorsJeffrey C. Miller, Edward J. Rebar, H. Steve Zhang
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Abstract
Disclosed herein are compositions comprising non-naturally occurring zinc finger domains, fusion proteins comprising these zinc finger domains, polynucleotides encoding these proteins, cells expressing these proteins and pharmaceutical compositions comprising these proteins or polynucleotides as well as methods of modifying an Htt gene using these compositions for treating or preventing Huntington's Disease.
Claims9
A non-naturally occurring TALE transcription factor (TALE-TF) that binds to an Htt gene, the TALE-TF comprising a fusion protein comprising a TALE DNA binding domain linked to a transcriptional repression domain, wherein the TALE DNA binding domain comprises an amino acid sequence of any one of SEQ ID NOs:
- 197, 198, or 199, wherein the transcriptional repression domain comprises an amino acid sequence of any one of SEQ ID NOs:
- 210, 211, or 212, and wherein the TALE-TF specifically binds to a mutant Huntington's gene allele (mHtt) comprising a nucleic acid sequence of any one of SEQ ID NOs:
- 200-207.
Dependencies are inferred from the claim text.