Therapeutic targeting of a long non-coding rna


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Access through your institution Buy or subscribe Antisense oligonucleotides (ASOs) against the long non-coding RNA UBE3A antisense transcript (_UBE3A-ATS_) could represent a feasible therapy


for the monogenic disorder Angelman syndrome, a new study reports. The disease results from loss of expression of the maternal _UBE3A_ allele in the presence of the imprinted (that is,


silenced) paternal allele. Meng _et al_. were able to activate expression of the paternal _Ube3a_ allele in cultured mouse neurons and in live mice by specifically targeting the silencing


_Ube3a-ATS_ with ASOs. Restoration of UBE3A protein in a mouse model of Angelman syndrome was sufficient to ameliorate cognitive deficits. Given that genomic organization and regulation at


the imprinting control centre is highly conserved between mice and humans, the researchers posit that their findings highlight a viable therapeutic strategy in humans. This is a preview of


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ADDITIONAL ACCESS OPTIONS: * Log in * Learn about institutional subscriptions * Read our FAQs * Contact customer support REFERENCES * Meng, L. et al. Towards a therapy for Angelman syndrome


by targeting a long non-coding RNA. _Nature_ http://dx.doi.org/10.1038/nature13975 (2014) Download references Authors * Linda Koch View author publications You can also search for this


author inPubMed Google Scholar RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Koch, L. Therapeutic targeting of a long non-coding RNA. _Nat Rev Genet_


16, 2 (2015). https://doi.org/10.1038/nrg3879 Download citation * Published: 09 December 2014 * Issue Date: January 2015 * DOI: https://doi.org/10.1038/nrg3879 SHARE THIS ARTICLE Anyone you


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