Published April 28, 2026
| Version v1
Preprint
Open
FMR1-AS1 Antisense Oligonucleotide Therapy
Description
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.
Notes
IPFS: QmRXfCKihJxodn5r5KLXJ9XqLr7rm3JZv8a6M2d3Y2m6YQ. TX: 0x7b7a363e9431d3fb8a9716a1b184b7279e64c3d8c937d6cd99a21861bde3dae7. CC0.
Files
hypothesis.json
Files
(324 Bytes)
| Name | Size | Download all |
|---|---|---|
|
md5:4790ff23fcf73ecdf47efb418827a3d6
|
324 Bytes | Preview Download |