---
title: "Runx1 Drives Cyst Growth in ADPKD: Inhibition with Ro5-3335 Slows Disease Progression"
id: "pubmed-42640700"
canonical_url: "https://medichelpline.com/clinical-feed/pubmed-42640700"
content_type: "clinical_feed_article"
specialty: "Nephrology"
source_name: "PubMed / NCBI"
source_url: "https://pubmed.ncbi.nlm.nih.gov/42640700/"
doi: "10.1096/fj.202600741R"
published_at: "2026-08-31T00:00:00.000Z"
evidence_level: "Journal Article"
license: "CC-BY-NC-4.0 / Informational Use"
---
# Runx1 Drives Cyst Growth in ADPKD: Inhibition with Ro5-3335 Slows Disease Progression
## Provenance & Clinical Metadata
- **Canonical URL:** https://medichelpline.com/clinical-feed/pubmed-42640700
- **Specialty:** [Nephrology](https://medichelpline.com/clinical-feed/nephrology.md)
- **Primary Source:** PubMed / NCBI
- **Source URL:** [Original Journal Publication](https://pubmed.ncbi.nlm.nih.gov/42640700/)
- **DOI:** [10.1096/fj.202600741R](https://doi.org/10.1096%2Ffj.202600741R)
- **Published At:** 2026-08-31T00:00:00.000Z
- **Evidence Rating:** Journal Article
## Executive GIST (TL;DR)
- Autosomal dominant polycystic kidney disease (**ADPKD**) features progressive kidney cyst formation that destroys renal parenchyma. The transcription factor **Runx1** is identified as a key driver of cyst growth in ADPKD. - Runx1 expression is markedly increased in **Pkd1** mutant renal epithelial cells and in kidneys from Pkd1 mutant mice. Upregulation occurs at both mRNA and protein levels and is evident in multiple renal epithelial cell populations. - Runx1 induction is mediated by **cAMP/PKA signaling**: Runx1 protein and mRNA increase with forskolin or constitutively active PKA and decrease with PKA inhibition or constitutive PKA suppression in Pkd1 models. - Pharmacologic inhibition of Runx1 using the selective inhibitor **Ro5-3335** significantly reduces cyst progression in both rapidly progressive (Pkd1 nl/nl) and slowly progressive (Pkd1 RC/RC) mouse models. - Ro5-3335 treatment reduced cystic index and kidney-weight-to-body-weight ratios and improved blood urea nitrogen (BUN) levels; Ki67 staining showed decreased epithelial proliferation. Reported group sizes included n = 9 per treatment in the cited experiments and several endpoints reached p < 0.01 or p < 0.05. - Mechanistically, Runx1 promotes cyst-lining epithelial cell proliferation by activating **AKT-mTOR**, **MAPK/ERK**, and **STAT3** signaling cascades and by suppressing **p53**-mediated apoptosis. - Runx1 also augments inflammatory and fibrotic responses: it promotes macrophage infiltration and NF-κB activation and aggravates interstitial fibrosis through **TGF-β/Smad2** signaling. - Collectively, the data identify Runx1 as a pivotal regulator of cyst growth and a potential therapeutic target in ADPKD; Ro5-3335 is presented as a candidate Runx1 inhibitor that slows cyst progression in preclinical Pkd1 mouse models. - Conflict of interest: authors declare no conflicts. Full experimental details, dosing regimens, and longer-term outcome data are available in the source article; any details not explicitly reported there are not inferred here.
## Clinical Analysis & Structured Key Points
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more resources ](https://pubmed.ncbi.nlm.nih.gov/42640700/#linkout) Title & authors Abstract Conflict of interest statement Figures Similar articles References MeSH terms Substances Related information Grants and funding LinkOut - more resources FASEB J Actions * [ Search in PubMed ](https://pubmed.ncbi.nlm.nih.gov/?term=%22FASEB+J%22%5Bjour%5D&sort=date&sort_order=desc) * [ Search in NLM Catalog ](https://www.ncbi.nlm.nih.gov/nlmcatalog?term=%22FASEB+J%22%5BTitle+Abbreviation%5D) * [ Add to Search ](https://pubmed.ncbi.nlm.nih.gov/42640700/) . 2026 Aug 31;40(16):e72209. doi: 10.1096/fj.202600741R. # Targeting Runx1 Slows Cyst Growth in Autosomal Dominant Polycystic Kidney Disease [Yueyue Zhang](https://pubmed.ncbi.nlm.nih.gov/?term=Zhang+Y&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.")[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-2 "Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA.")[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-3 "Department of Nephrology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China."), [Chang Liu](https://pubmed.ncbi.nlm.nih.gov/?term=Liu+C&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Junchi Liu](https://pubmed.ncbi.nlm.nih.gov/?term=Liu+J&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Linda Xiaoyan Li](https://pubmed.ncbi.nlm.nih.gov/?term=Li+LX&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Vicente Torres](https://pubmed.ncbi.nlm.nih.gov/?term=Torres+V&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Xiaogang Li](https://pubmed.ncbi.nlm.nih.gov/?term=Li+X&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.")[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-2 "Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA."), [Julie Xia Zhou](https://pubmed.ncbi.nlm.nih.gov/?term=Zhou+JX&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#full-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.") Affiliations Expand ### Affiliations * 1 Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA. * 2 Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA. * 3 Department of Nephrology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China. * PMID: **42640700** * PMCID: [ PMC13505735 ](https://pmc.ncbi.nlm.nih.gov/articles/PMC13505735/) * DOI: [ 10.1096/fj.202600741R ](https://doi.org/10.1096/fj.202600741r) Item in Clipboard # Targeting Runx1 Slows Cyst Growth in Autosomal Dominant Polycystic Kidney Disease Yueyue Zhang et al. FASEB J. 2026. Show details Display options Display options Format Abstract PubMed PMID FASEB J Actions * [ Search in PubMed ](https://pubmed.ncbi.nlm.nih.gov/?term=%22FASEB+J%22%5Bjour%5D&sort=date&sort_order=desc) * [ Search in NLM Catalog ](https://www.ncbi.nlm.nih.gov/nlmcatalog?term=%22FASEB+J%22%5BTitle+Abbreviation%5D) * [ Add to Search ](https://pubmed.ncbi.nlm.nih.gov/42640700/) . 2026 Aug 31;40(16):e72209. doi: 10.1096/fj.202600741R. ### Authors [Yueyue Zhang](https://pubmed.ncbi.nlm.nih.gov/?term=Zhang+Y&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.")[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-2 "Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA.")[ 3 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-3 "Department of Nephrology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China."), [Chang Liu](https://pubmed.ncbi.nlm.nih.gov/?term=Liu+C&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Junchi Liu](https://pubmed.ncbi.nlm.nih.gov/?term=Liu+J&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Linda Xiaoyan Li](https://pubmed.ncbi.nlm.nih.gov/?term=Li+LX&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Vicente Torres](https://pubmed.ncbi.nlm.nih.gov/?term=Torres+V&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA."), [Xiaogang Li](https://pubmed.ncbi.nlm.nih.gov/?term=Li+X&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.")[ 2 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-2 "Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA."), [Julie Xia Zhou](https://pubmed.ncbi.nlm.nih.gov/?term=Zhou+JX&cauthor_id=42640700)[ 1 ](https://pubmed.ncbi.nlm.nih.gov/42640700/#short-view-affiliation-1 "Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.") ### Affiliations * 1 Division of Nephrology and Hypertension, Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA. * 2 Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota, USA. * 3 Department of Nephrology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China. * PMID: **42640700** * PMCID: [ PMC13505735 ](https://pmc.ncbi.nlm.nih.gov/articles/PMC13505735/) * DOI: [ 10.1096/fj.202600741R ](https://doi.org/10.1096/fj.202600741r) Item in Clipboard Full text links Cite Display options Display options Format Abstract PubMed PMID ## Abstract Autosomal dominant polycystic kidney disease (ADPKD), the most prevalent hereditary kidney disorder, is characterized by the progressive formation and expansion of kidney cysts that ultimately destroy normal renal parenchyma. Runt-related transcription factor 1 (Runx1), a highly conserved regulator of gene expression, orchestrates diverse cellular signaling pathways. Here, we identify Runx1 as a critical driver of cyst growth in ADPKD. Runx1 expression is markedly upregulated in Pkd1 mutant renal epithelial cells and kidney tissues through a cAMP-dependent mechanism. Pharmacological inhibition of Runx1 with its selective inhibitor, Ro5-3335, significantly attenuates cyst progression in both rapidly and slowly progressive Pkd1 mouse models. Mechanistically, Runx1 enhances proliferation of cyst-lining epithelial cells by activating the AKT-mTOR, MAPK, and STAT3 signaling cascades, while suppressing p53-mediated apoptosis. Moreover, Runx1 promotes macrophage infiltration and inflammatory responses via NF-κB activation and aggravates interstitial fibrosis through the TGF-β/Smad2 pathway. Collectively, these findings uncover Runx1 as a pivotal regulator of cystic disease progression and highlight it as a promising therapeutic target for ADPKD. © 2026 Federation of American Societies for Experimental Biology. [PubMed Disclaimer](https://pubmed.ncbi.nlm.nih.gov/disclaimer/) ## Conflict of interest statement The authors declare no conflicts of interest. ## Figures [ ![FIGURE 1](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/25a0bc5b441c/FSB2-40-e72209-g007.gif) ](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/ae8373535309/FSB2-40-e72209-g007.webp) ** FIGURE 1 ** Runx1 is upregulated in _Pkd1_ … ** FIGURE 1 ** Runx1 is upregulated in _Pkd1_ mutant renal epithelial cells and kidney tissues. (A,… **FIGURE 1** Runx1 is upregulated in _Pkd1_ mutant renal epithelial cells and kidney tissues. (A, B) Runx1 mRNA (A) and protein (B) expression in mouse embryonic _Pkd1_ wild‐type cells (WT) and _Pkd1_ null MEK cells (Null). _p_ < 0.05. (C, D) Runx1 mRNA (C) and protein (D) expression in kidneys from PN21 _Pkd1_ _nl/nl_ mice and wild‐type (WT) mice. _p_ < 0.01. (E) Coimmunostaining of Runx1 (red) and LTL (green) in kidneys from PN21 WT mice and _Pkd1_ _nl/nl_ mice. (F) Coimmunostaining of Runx1 (red) and THP (green) in kidneys from three‐month WT mice and _Pkd1_ _nl/nl_ mice. (G) Coimmunostaining of Runx1 (red) and DBA (green) in kidneys from 3‐month‐old WT mice and _Pkd1_ _nl/nl_ mice. Nuclei were counterstained with DAPI (blue). Scale bars, 50 μm. [ ![FIGURE 2](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/9af78e9981ef/FSB2-40-e72209-g006.gif) ](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/b5a0d2865bdd/FSB2-40-e72209-g006.webp) ** FIGURE 2 ** Runx1 expression is activated by… ** FIGURE 2 ** Runx1 expression is activated by cAMP signaling. (A) Runx1 protein expression in mouse… **FIGURE 2** Runx1 expression is activated by cAMP signaling. (A) Runx1 protein expression in mouse embryonic _Pkd1_ wild‐type cells (WT) and PH2 cells treated with forskolin. (B) Runx1 protein expression in _Pkd1_ null MEK cells (Null) and PN24 cells treated with the PKA inhibitor H89. (C–E) Relative expression of Runx1 mRNA in kidneys from constitutively activated PKA (CA‐PKA) wild‐type versus control wild‐type mice (C, _p_ < 0.01), CA‐PKA _Pkd1_ _RC/RC_ versus _Pkd1_ _RC/RC_ mice (D, _p_ < 0.05), and constitutively inhibited PKA (CI‐PKA) _Pkd1_ _RC/RC_ versus _Pkd1_ _RC/RC_ mice (E, _p_ < 0.01.). [ ![FIGURE 3](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/6f02ca3a9732/FSB2-40-e72209-g001.gif) ](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/51fd3f45f72a/FSB2-40-e72209-g001.webp) ** FIGURE 3 ** Ro5‐3335 treatment slows cyst growth… ** FIGURE 3 ** Ro5‐3335 treatment slows cyst growth in _Pkd1_ _nl/nl_ and _Pkd1_ … **FIGURE 3** Ro5‐3335 treatment slows cyst growth in _Pkd1_ _nl/nl_ and _Pkd1_ _RC/RC_ mice. (A, B) Representative kidneys (A) and H&E staining of kidneys (B) from PN21 _Pkd1_ _nl/nl_ mice treated with vehicle (_n_ = 9) or Ro5‐3335 (_n_ = 9). Scale bar, 1 mm. (C–F) Cystic index (C, _p_ < 0.01), KW/BW ratios (D, _p_ < 0.01), body weight (E, _p_ = 0.17), and BUN levels (F, _p_ < 0.05) of PN21 _Pkd1_ _nl/nl_ mice treated with vehicle or Ro5‐3335. (G) Ki67 staining of kidneys from PN21 _Pkd1_ _nl/nl_ mice treated with vehicle or Ro5‐3335. Ki67, green; DAPI, blue. Scale bar, 20 μm. _p_ < 0.01. (H, I) Representative kidneys (H) and H&E staining of kidneys (I) from 3‐month‐old _Pkd1_ _RC/RC_ mice _treated with_ vehicle (_n_ = 9) or Ro5‐3335 (_n_ = 9). Scale bar, 1 mm. (J–M) Cystic index (J, _p_ < 0.01), KW/BW ratios (K, _p_ < 0.01), body weight (L, _p_ = 0.24), and BUN levels (M, _p_ < 0.01) of 3‐month‐old _Pkd1_ _RC/RC_ mice treated with vehicle or Ro5‐3335. (N) Ki67 staining of kidneys from 3‐month‐old _Pkd1_ _RC/RC_ mice treated with vehicle or Ro5‐3335. Ki67, green; DAPI, blue. Scale bar, 20 μm. _p_ < 0.01. [ ![FIGURE 4](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/b46e935ed199/FSB2-40-e72209-g004.gif) ](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/91af53955c93/FSB2-40-e72209-g004.webp) ** FIGURE 4 ** Runx1 activates PKD associated signaling… ** FIGURE 4 ** Runx1 activates PKD associated signaling pathways. (A, B) Phosphorylation levels of Akt, mTOR,… **FIGURE 4** Runx1 activates PKD associated signaling pathways. (A, B) Phosphorylation levels of Akt, mTOR, S6, ERK, and STAT3 in kidneys from PN21 _Pkd1_ _nl/nl_ mice treated with vehicle or Ro5‐3335 (A), and in kidneys from 3‐month‐old _Pkd1_ _RC/RC_ mice treated with vehicle or Ro5‐3335 (B). (C) Phosphorylation levels of Akt, mTOR, S6, ERK, and STAT3 in PN24 cells treated with control siRNA or Runx1 siRNA for 48 h. (D, E) Phosphorylation levels of Akt, mTOR, S6, ERK, and STAT3 in PN24 cells treated with Ro5‐3335 for 24 h at different concentrations (D) and with 40 μM Ro5‐3335 for different times as indicated (E). [ ![FIGURE 5](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/27795ac3fb3e/FSB2-40-e72209-g005.gif) ](https://cdn.ncbi.nlm.nih.gov/pmc/blobs/31f7/13505735/6b8c5871b3a0/FSB2-40-e72209-g005.webp) ** FIGURE 5 ** Runx1 regulates cystic renal epithelial… ** FIGURE 5 ** Runx1 regulates cystic renal epithelial cell death. (A) Treatment with Ro5‐3335 induced cyst‐lining… **F
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