Evidence map›Paper›PMID 40192175›Full record

ArticleAngewandte Chemie (International ed. in English)2025

New Transferrin Receptor-Targeting Conjugate Effectively Delivers DNA to Mouse Brain.

Min-Sun Song, Adrian H Bustos, Lise Bastue, Justas Mikutavicius, Piotr Swiderski, Kelly J Clemens, Nagy Habib, John Rossi, Kira Astakhova

Abstract read
In one paragraph

Article in Angewandte Chemie (International ed. in English), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Review
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

9 authors.

Min-Sun Song *Department of Molecular and Cellular Biology, City of Hope National Medical Center, City of Hope, 1500 East Duarte Road, Duarte, 91010, USA.
Adrian H Bustos *Department of Chemistry, Organic and Inorganic Chemistry, Technical University of Denmark, Kemitorvet, Building 207, Lyngby, 2800 Kgs, Denmark.
Lise BastueDepartment of Chemistry, Organic and Inorganic Chemistry, Technical University of Denmark, Kemitorvet, Building 207, Lyngby, 2800 Kgs, Denmark.
Justas MikutaviciusDepartment of Chemistry, Organic and Inorganic Chemistry, Technical University of Denmark, Kemitorvet, Building 207, Lyngby, 2800 Kgs, Denmark.
Piotr SwiderskiDepartment of Molecular and Cellular Biology, City of Hope National Medical Center, City of Hope, 1500 East Duarte Road, Duarte, 91010, USA.
Kelly J ClemensSchool of Psychology, Faculty of Science, University New South Wales, Kensington, New South Wales, 2052, Australia.
Nagy HabibDepartment of Surgery & Cancer, Imperial College London, Hammersmith Campus, Du Cane Road, London, W12 0NN, UK.
John RossiDepartment of Molecular and Cellular Biology, City of Hope National Medical Center, City of Hope, 1500 East Duarte Road, Duarte, 91010, USA.
Kira AstakhovaDepartment of Chemistry, Organic and Inorganic Chemistry, Technical University of Denmark, Kemitorvet, Building 207, Lyngby, 2800 Kgs, Denmark.

Funding

Transgenic Mouse FacilityP30CA033572 · NCI · CITY OF HOPE/BECKMAN RESEARCH INSTITUTE · PI John Charles Williams · 1985 to 2026
$86.3M
City of HopeDTU P30CA33572NCI NIH HHS P30 CA033572
6 · The paper itself

Abstract

Delivery across the blood-brain barrier (BBB) is one of the most challenging tasks for modern biopharmaceutics. Many attempts have been taken, with only low delivery efficacies achieved so far. We report a new transferrin receptor-targeting (TfR) RNA aptamer conjugated to DSPE lipid that leads to an unprecedented effective uptake in the brain, with brain-to-serum ratios up to 6.5 in mice. This result is superior to recently published values of < 1 for antibody conjugates and nanovesicles, pointing to a successful combined effect of the increased lipophilicity and TfR targeting with the new RNA aptamer that our conjugate provides. Using fluorescence whole body imaging, polymerase-chain reaction (PCR) and fluorescence in situ hybridization, we confirm that the new conjugate delivers high amounts of DNA oligonucleotide to brains of Balb/cJ mice, and it is effective in human cells. There is no acute toxicity as verified with histopathological assessment of mice organs. The combination of properties demonstrated by our new conjugate makes it a highly potent delivery tool that can be applied in therapy of brain diseases incl. glioblastoma, neurogenerative diseases, and a broad range of brain infections.

Indexed as

Aptamers, NucleotideBrainDNAReceptors, TransferrinAnimalsBlood-Brain BarrierDrug Delivery SystemsHumansMiceMice, Inbred BALB CAptamers, NucleotideDNAReceptors, TransferrinAnti‐miRAptamerBBBNucleic acid deliveryTransferrin receptor

Identifiers

PMID40192175
PMCPMC12144868

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.