Evidence map›Paper›PMID 28412169›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017

Engineered Exosomes as Vehicles for Biologically Active Proteins.

Ulrich Sterzenbach, Ulrich Putz, Ley-Hian Low, John Silke, Seong-Seng Tan, Jason Howitt

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 203 papers.

0numbers the graph read from it
0cells of the map it votes in
203citing papers in PubMed
10.7field-weighted citation impact, top 1% of its field
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

203 citing papers in PubMed, 332 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Endogenous Engineering Reprograms Extracellular Vesicles for Enhanced Therapeutic Function.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Review
  5. Article
  6. Review
  7. Review
  8. Preclinical assessment of genetically modified exosomes for colorectal cancer immunotherapy.Journal of controlled release : official journal of the Controlled Release Society · 2026
    Article
  9. Review
  10. Review
  11. Review
  12. Exosomes as Emerging Nanocarriers for Targeted Cancer Therapy.International journal of nanomedicine · 2026
    Review
  13. Review
  14. Article
  15. Article
  16. Review
  17. Review
  18. Article
  19. Review
  20. Review

143 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 2 countries.

Ulrich SterzenbachThe Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia.
Ulrich PutzThe Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia.
Ley-Hian LowUniversity of California San Francisco and Veterans Affairs Medical Center, San Francisco, CA 94121, USA.
John SilkeWalter and Eliza Hall Institute, The University of Melbourne, Parkville, Victoria 3052, Australia.
Seong-Seng TanThe Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia.
Jason HowittThe Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria 3052, Australia. Electronic address: jason.howitt@florey.edu.au.
University of Melbourne · AUFlorey Institute of Neuroscience and Mental Health · AUUniversity of California, San Francisco · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Exosomes represent an attractive vehicle for the delivery of biomolecules. However, mechanisms for loading functional molecules into exosomes are relatively unexplored. Here we report the use of the evolutionarily conserved late-domain (L-domain) pathway as a mechanism for loading exogenous proteins into exosomes. We demonstrate that labeling of a target protein, Cre recombinase, with a WW tag leads to recognition by the L-domain-containing protein Ndfip1, resulting in ubiquitination and loading into exosomes. Our results show that Ndfip1 expression acts as a molecular switch for exosomal packaging of WW-Cre that can be suppressed using the exosome inhibitor GW4869. When taken up by floxed reporter cells, exosomes containing WW-Cre were capable of inducing DNA recombination, indicating functional delivery of the protein to recipient cells. Engineered exosomes were administered to the brain of transgenic reporter mice using the nasal route to test for intracellular protein delivery in vivo. This resulted in the transport of engineered exosomes predominantly to recipient neurons in a number of brain regions, including the olfactory bulb, cortex, striatum, hippocampus, and cerebellum. The ability to engineer exosomes to deliver biologically active proteins across the blood-brain barrier represents an important step for the development of therapeutics to treat brain diseases.

Indexed as

Drug Delivery SystemsGenetic EngineeringProtein TransportAnimalsBlood-Brain BarrierBrainCarrier ProteinsCell LineExosomesExtracellular VesiclesGene ExpressionIntegrasesMembrane ProteinsMiceNasal AbsorptionPermeabilityCarrier ProteinsCre recombinaseIntegrasesMembrane ProteinsNDFIP1 protein, humanRecombinant Fusion Proteinsblood-brain barrierdrug deliveryESCRTextracellular vesiclesintraluminal vesiclesL-domainnasal deliverytherapyubiquitin

Identifiers

PMID28412169
PMCPMC5474961
OpenAlexW2605222169

What OpenQuestion holds

Textmetadata
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.