EPIGENETIC MODULATION OF GENOMIC TARGETS TO CONTROL EXPRESSION OF PWS-ASSOCIATED GENES

摘要

Disclosed herein are DNA targeting systems that target a regulatory element of a gene within the 15q11-13 locus. Further provided are DNA targeting systems including at least one gRNA and a Cas9 protein, as well as compositions comprising the same. The compositions may be used in methods for treating Prader-Willi Syndrome (PWS) in a subject. The method may include administering to a subject the DNA targeting system.