Evidence map›Paper›PMID 38956234›Full record

ArticleScientific reports2024

Rapamycin-encapsulated nanoparticle delivery in polycystic kidney disease mice.

Shinobu Yamaguchi, Randee Sedaka, Chintan Kapadia, Jifeng Huang, Jung-Shan Hsu, Taylor F Berryhill, Landon Wilson, Stephen Barnes, Caleb Lovelady, Yasin Oduk and 4 more

Abstract read
In one paragraph

Article in Scientific reports, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Review
  4. Combination siRNA delivery as a therapeutic strategy for ADPKD.bioRxiv : the preprint server for biology · 2026
    Article
  5. Review
  6. Article
  7. Nanotherapeutics in Kidney Disease: Innovations, Challenges, and Future Directions.Journal of the American Society of Nephrology : JASN · 2025
    Review
  8. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

14 authors.

Shinobu Yamaguchi *Division of Nephrology, Department of Medicine, Section of Cardio-Renal Physiology and Medicine, McCallum Basic Health Science Building, University of Alabama at Birmingham, Room 533, 1918 University Blvd, Birmingham, AL, 35233, USA.
Randee Sedaka *Division of Nephrology, Department of Medicine, Section of Cardio-Renal Physiology and Medicine, McCallum Basic Health Science Building, University of Alabama at Birmingham, Room 533, 1918 University Blvd, Birmingham, AL, 35233, USA.
Chintan KapadiaGoldilocks Therapeutics, Inc., Bedford, NY, USA.
Jifeng HuangDivision of Nephrology, Department of Medicine, Section of Cardio-Renal Physiology and Medicine, McCallum Basic Health Science Building, University of Alabama at Birmingham, Room 533, 1918 University Blvd, Birmingham, AL, 35233, USA.
Jung-Shan HsuDivision of Nephrology, Department of Medicine, Section of Cardio-Renal Physiology and Medicine, McCallum Basic Health Science Building, University of Alabama at Birmingham, Room 533, 1918 University Blvd, Birmingham, AL, 35233, USA.
Taylor F BerryhillTargeted Metabolomics and Proteomics Laboratory, University of Alabama at Birmingham, Birmingham, AL, USA.
Landon WilsonTargeted Metabolomics and Proteomics Laboratory, University of Alabama at Birmingham, Birmingham, AL, USA.
Stephen BarnesTargeted Metabolomics and Proteomics Laboratory, University of Alabama at Birmingham, Birmingham, AL, USA.
Caleb LoveladyDivision of Nephrology, Department of Medicine, Section of Cardio-Renal Physiology and Medicine, McCallum Basic Health Science Building, University of Alabama at Birmingham, Room 533, 1918 University Blvd, Birmingham, AL, 35233, USA.
Yasin OdukNanomediGene LLC, Birmingham, AL, USA.
Ryan M WilliamsDepartment of Biomedical Engineering, The City College of New York, New York, NY, USA.
Edgar A JaimesDepartment of Medicine, Renal Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Daniel A HellerMolecular Pharmacology Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Takamitsu SaigusaDivision of Nephrology, Department of Medicine, Section of Cardio-Renal Physiology and Medicine, McCallum Basic Health Science Building, University of Alabama at Birmingham, Room 533, 1918 University Blvd, Birmingham, AL, 35233, USA. tsaigusa@uabmc.edu.

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
XRAY CRYSTALLOGRAPHYP30CA013148 · NCI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Robert S Welner · 1985 to 2026
$165.9M
Research Center in Minority Institutions (RCMI) at City CollegeU54MD017979 · NIMHD · CITY COLLEGE OF NEW YORK · PI M. Felice Marina GHILARDI · 2024 to 2026
$15.9M
TRAINING IN CELLULAR PHYSIOLOGY MEMBRANE TRANSPORTT32DK007545 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI GUTIERREZ, ORLANDO M · 1987 to 2023
$4.5M
Nanotechnology as a therapeutic approach in arteriovenous fistula maturationR01DK129299 · NIDDK · UNIVERSITY OF UTAH · PI JAIMES, EDGAR A, SHIU, YAN-TING E. · 2021 to 2024
$1.7M
The Role of Wheat-Gluten in Polycystic Kidney DiseaseR01DK132028 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI Takamitsu Saigusa · 2022 to 2026
$1.5M
Renal tubule-specific nanotherapeutics for acute kidney injuryR01DK114321 · NIDDK · SLOAN-KETTERING INST CAN RESEARCH · PI HELLER, DANIEL ALAN, JAIMES, EDGAR A · 2018 to 2020
$796k
The Role of Kidney Immune Cells in Acceleration of Cyst GrowthR03DK119717 · NIDDK · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI SAIGUSA, TAKAMITSU · 2019 to 2020
$223k
Congressionally Directed Medical Research Programs W81XWH-21-1-0188NCI NIH HHS P30 CA008748NCI NIH HHS P30 CA013148NIDDK NIH HHS DK007545NIDDK NIH HHS DK114321NIDDK NIH HHS DK119717NIDDK NIH HHS DK129299NIDDK NIH HHS DK132028NIDDK NIH HHS R01 DK132028NIDDK NIH HHS R03 DK119717NIDDK NIH HHS T32 DK007545NIH HHS P30 CA008748NIMHD NIH HHS U54 MD017979PKDF 232G19a
6 · The paper itself

Abstract

Rapamycin slows cystogenesis in murine models of polycystic kidney disease (PKD) but failed in clinical trials, potentially due to insufficient drug dosing. To improve drug efficiency without increasing dose, kidney-specific drug delivery may be used. Mesoscale nanoparticles (MNP) selectively target the proximal tubules in rodents. We explored whether MNPs can target cystic kidney tubules and whether rapamycin-encapsulated-MNPs (RapaMNPs) can slow cyst growth in Pkd1 knockout (KO) mice. MNP was intravenously administered in adult Pkd1KO mice. Serum and organs were harvested after 8, 24, 48 or 72 h to measure MNP localization, mTOR levels, and rapamycin concentration. Pkd1KO mice were then injected bi-weekly for 6 weeks with RapaMNP, rapamycin, or vehicle to determine drug efficacy on kidney cyst growth. Single MNP injections lead to kidney-preferential accumulation over other organs, specifically in tubules and cysts. Likewise, one RapaMNP injection resulted in higher drug delivery to the kidney compared to the liver, and displayed sustained mTOR inhibition. Bi-weekly injections with RapaMNP, rapamycin or vehicle for 6 weeks resulted in inconsistent mTOR inhibition and little change in cyst index, however. MNPs serve as an effective short-term, kidney-specific delivery system, but long-term RapaMNP failed to slow cyst progression in Pkd1KO mice.

Indexed as

Disease Models, AnimalMice, KnockoutNanoparticlesPolycystic Kidney DiseasesSirolimusAnimalsDrug Delivery SystemsKidneyMaleMiceTOR Serine-Threonine KinasesTRPP Cation ChannelsSirolimusTOR Serine-Threonine KinasesTRPP Cation Channels

Identifiers

PMID38956234
PMCPMC11219830

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.