RGLS8429-Mediated miR-17 Inhibition Leads to Acute PKD1/2 De-repression and Ameliorates Preclinical ADPKD: TH-PO435
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R Lakhia, JA Alvarez, H Ramalingam…
Journal of the …, 2024
journals.lww.com
Background: ADPKD is caused by reduced PKD1/2 gene dosage. However, drugs that improve PKD1/2 levels are not available. We have shown that microRNA-17 directly represses PKD1/2 and aggravates ADPKD progression. Thus, our goals were to characterize the next-gen anti-miR-17 drug RGLS8429 and its impact on PKD1/2 and ADPKD. Methods: We treated murine and human ADPKD kidney cell lines and mouse models with PBS, control oligo, or RGLS8429 to assess for miR-17 inhibition, PKD1/2 de …
