Evidence map›Paper›PMID 40963409›Full record

ArticleJournal of cell science2025

Lipid nanoparticle-encapsulated Dnai1 mRNA rescues ciliary activity in primary ciliary dyskinesia mouse cell models.

Amanda J Smith, Patrick R Sears, Mirko Hennig, Rumpa B Bhattacharjee, Weining Yin, Hannah Golliher, Daniella Ishimaru, T Noelle Lombana, David J Lockhart, Brandon A Wustman and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of cell science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–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

2 citing papers in PubMed.

  1. Preclinical human models of primary ciliary dyskinesia.European respiratory review : an official journal of the European Respiratory Society · 2026
    Review
  2. 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

11 authors.

Amanda J SmithMarsico Lung Institute/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.ORCID 0000-0002-3693-7917
Patrick R SearsMarsico Lung Institute/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Mirko HennigReCode Therapeutics, Menlo Park, CA 94025, USA.ORCID 0009-0009-0238-737X
Rumpa B BhattacharjeeReCode Therapeutics, Menlo Park, CA 94025, USA.
Weining YinMarsico Lung Institute/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Hannah GolliherMarsico Lung Institute/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Daniella IshimaruReCode Therapeutics, Menlo Park, CA 94025, USA.ORCID 0009-0009-3443-6869
T Noelle LombanaReCode Therapeutics, Menlo Park, CA 94025, USA.
David J LockhartReCode Therapeutics, Menlo Park, CA 94025, USA.
Brandon A WustmanReCode Therapeutics, Menlo Park, CA 94025, USA.
Lawrence E OstrowskiMarsico Lung Institute/Cystic Fibrosis Research and Treatment Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.ORCID 0000-0002-7558-6603

Funding

ReCode TherapeuticsUniversity of North Carolina at Chapel Hill
6 · The paper itself

Abstract

Primary ciliary dyskinesia (PCD) is a rare, genetically heterogenous disorder resulting from dysfunctional motile cilia that is characterized by chronic, progressive lung disease with currently no corrective therapies available. Here, we test the efficacy of selective organ targeting lipid nanoparticles (SORT-LNPs) that were optimized for potency and delivery to respiratory cells containing an mRNA encoding an axonemal protein to rescue ciliary activity in a murine culture model of PCD. Utilizing murine nasopharyngeal epithelial cell (mNPEC) cultures isolated from a conditional Dnai1 knockout mouse model of the known human PCD-associated gene DNAI1 homolog, we tested if SORT-LNPs containing an optimized Dnai1 mRNA could rescue ciliary activity. Treatment of differentiating and well-differentiated Dnai1 knockout mNPECs with SORT-LNP-Dnai1 mRNA led to a dose-dependent increase in levels of DNAI1 protein and incorporation into ciliary axonemes, resulting in rescued ciliary activity with normal ciliary beat frequency that persisted for over 3 weeks. These data support further clinical development of an mRNA-based therapeutic with LNP-mediated delivery as a treatment for individuals with PCD with disease-causing DNAI1 mutations.

Indexed as

CiliaCiliary Motility DisordersLipidsNanoparticlesRNA, MessengerAnimalsDisease Models, AnimalEpithelial CellsHumansLiposomesMiceMice, KnockoutTumor Suppressor ProteinsLipid NanoparticlesLipidsLiposomesRNA, MessengerTg737Rpw protein, mouseTumor Suppressor ProteinsLipid nanoparticlesMotile ciliamRNA therapyPrimary ciliary dyskinesia

Identifiers

PMID40963409
PMCPMC12633742

What OpenQuestion holds

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Registered trials

None linked

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.