Methods and compositions for treating trinucleotide repeat disorders
First Claim
Patent Images
1. A cell comprising a complex of a non-naturally occurring zinc finger protein transcription factor and a target site in an Htt gene, wherein the zinc finger protein transcription factor comprises a transcriptional repression domain and a finger protein comprising 5 or 6 zinc finger recognition regions, ordered F1 to F5 or F1 to F6 from N-terminus to C-terminus, wherein the zinc finger protein comprises the recognition helix regions in the order shown in a single row of Table 1 bound to the target site in the Htt gene, the target site comprising sequences as set forth in any of SEQ ID NO:
- 20-23 and 36-39 and further wherein Htt gene expression is repressed in the cell.
1 Assignment
0 Petitions
Accused Products
Abstract
Disclosed herein are methods and compositions for treating trinucleotide repeat disorders.
52 Citations
8 Claims
-
1. A cell comprising a complex of a non-naturally occurring zinc finger protein transcription factor and a target site in an Htt gene, wherein the zinc finger protein transcription factor comprises a transcriptional repression domain and a finger protein comprising 5 or 6 zinc finger recognition regions, ordered F1 to F5 or F1 to F6 from N-terminus to C-terminus, wherein the zinc finger protein comprises the recognition helix regions in the order shown in a single row of Table 1 bound to the target site in the Htt gene, the target site comprising sequences as set forth in any of SEQ ID NO:
- 20-23 and 36-39 and further wherein Htt gene expression is repressed in the cell.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8)
Specification