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- W3217492735 abstract "Abstract In imprinting disorders, where the active copy of an imprinted gene is mutated or lost, there is a unique opportunity for causal treatment by unsilencing the other, dormant allele. Depending on the mechanism by which the allele is silenced, unsilencing can be achieved by epigenetic drugs, antisense-oligonucleotides (ASOs) or epigenome editing. While most of the research is still preclinical, first-in-humans studies with ASOs have started in 2020." @default.
- W3217492735 created "2021-12-06" @default.
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- W3217492735 date "2020-11-18" @default.
- W3217492735 modified "2023-09-27" @default.
- W3217492735 title "Novel strategies to cure imprinting disorders" @default.
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- W3217492735 doi "https://doi.org/10.1515/medgen-2020-2043" @default.
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