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FMR1-AS1 Antisense Oligonucleotide Therapy

descriptionPublicationkeyboard_double_arrow_right Preprint Under curationPublisher:Zenodo
Authors: MedicOath Autonomous Discovery Engine;

doi: 10.5281/zenodo.19832902

FMR1-AS1 Antisense Oligonucleotide Therapy

Abstract

FMR1-AS1 (antisense RNA) shows 0.39 association with FXS in Open Targets, second only to FMR1 itself. As an antisense transcript, it may regulate FMR1 expression. Targeted antisense oligonucleotides could modulate FMR1-AS1 to increase residual FMR1 protein production in patients with hypermethylated but not deleted FMR1 genes.

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