METHODS AND COMPOSITIONS FOR TREATING HUNTINGTON'S DISEASE
摘要
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.
申请公布号
EP2820159(A4)
申请公布日期
2015.08.26
申请号
EP20130755872
申请日期
2013.02.28
申请人
SANGAMO BIOSCIENCES, INC.
发明人
MILLER, JEFFREY, C.;REBAR, EDWARD, J.;ZHANG, H., STEVE