| abstract |
Disclosed herein are methods and compositions for treating Huntington's disease. In particular, provided herein are methods and compositions for modifying the HD Htt allele (eg, modulating its expression) to treat Huntington's disease. Methods and compositions for generating an animal model of Huntington's disease are also provided. Thus, in one embodiment, an engineered (non-naturally occurring) DNA binding domain (eg, zinc finger protein, TAL effector (TALE) protein or CRISPR / dCas − that regulates expression of an HD allele (eg, Htt) TF) is provided. |