| Abstrakt: |
"In some embodiments, the target gene enhancer sequence comprises two or more alleles and the enhancer-targeting aTF comprises a programmable DNA binding domain specific for a subset of the alleles; and/or the target gene promoter sequence comprises two or more alleles and the promoter-targeting aTF comprises a programmable DNA binding domain specific for a subset of the alleles. The aTF system of claim 2, wherein the enhancer-targeting aTF, the promoter-targeting aTF, or both each comprises two or more gene expression modulating domains. "In some embodiments, the promoter-targeting aTF(s) comprise (a) a fusion protein comprising a catalytically inactive Cas9 or catalytically inactive Cpf1 and a gene expression modulating domain; and (b) a gRNA comprising a sequence complementary to a target gene promoter sequence. [Extracted from the article] |
