US Patent:
20150225717, Aug 13, 2015
Inventors:
- Boston MA, US
Stefan Pinter - Cambridge MA, US
Barry Kesner - Charlestown MA, US
International Classification:
C12N 15/113
Abstract:
Methods and compositions for selectively reactivating genes on the inactive X chromosome (Xi) in a locus-specific manner, e.g., genes associated with X-linked diseases, e.g., Rett Syndrome, Factor VIII or IX deficiency, Fragile X Syndrome, Duchenne muscular dystrophy, and PNH, in heterozygous females.
