Ontology highlight
ABSTRACT:
SUBMITTER: Fimiani C
PROVIDER: S-EPMC5172352 | biostudies-literature | 2016 Dec
REPOSITORIES: biostudies-literature
Fimiani Cristina C Goina Elisa E Su Qin Q Gao Guangping G Mallamaci Antonello A
Scientific reports 20161220
More than one hundred distinct gene hemizygosities are specifically linked to epilepsy, mental retardation, autism, schizophrenia and neuro-degeneration. Radical repair of these gene deficits via genome engineering is hardly feasible. The same applies to therapeutic stimulation of the spared allele by artificial transactivators. Small activating RNAs (saRNAs) offer an alternative, appealing approach. As a proof-of-principle, here we tested this approach on the Rett syndrome-linked, haploinsuffic ...[more]