Evidence map›Paper›PMID 36698310›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2023

Extracellular vesicles: The next generation in gene therapy delivery.

Riccardo Cecchin, Zach Troyer, Ken Witwer, Kevin V Morris

Abstract readReview
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 120 papers.

0numbers the graph read from it
0cells of the map it votes in
120citing 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

120 citing papers in PubMed.

  1. Review
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  13. RPS3-Enriched Extracellular Vesicles Mediate Liver-Spinal Cord Inter-Organ Communication.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Article
  14. Review
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  16. In Vivo CAR-T Therapies-A New Era of Programmable Immunity.International journal of molecular sciences · 2026
    Review
  17. Article
  18. Article
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  20. Article

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

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

4 authors.

Riccardo CecchinMenzies Health Institute Queensland, School of Pharmacy and Medical Science, Griffith University, Gold Coast Campus, Southport, QLD 4222, Australia.
Zach TroyerDepartments of Molecular and Comparative Pathobiology and Neurology, and Richman Family Precision Medicine Center of Excellence in Alzheimer's Disease, Johns Hopkins University, Baltimore, MD 21205, USA.
Ken WitwerDepartments of Molecular and Comparative Pathobiology and Neurology, and Richman Family Precision Medicine Center of Excellence in Alzheimer's Disease, Johns Hopkins University, Baltimore, MD 21205, USA. Electronic address: kwitwer1@jhmi.edu.
Kevin V MorrisMenzies Health Institute Queensland, School of Pharmacy and Medical Science, Griffith University, Gold Coast Campus, Southport, QLD 4222, Australia. Electronic address: kevin.morris@griffith.edu.au.

Funding

Next-generation extracellular vesicle biologics to target central nervous system and peripheral reservoirs of HIVR01AI144997 · NIAID · JOHNS HOPKINS UNIVERSITY · PI RAMRATNAM, BHARAT, WITWER, KENNETH W · 2019 to 2023
$4.0M
Extracellular Vesicle and Extracellular RNA Biomarkers of HIV-1 Central Nervous System Pathogenesis and Cigarette UseR01DA047807 · NIDA · JOHNS HOPKINS UNIVERSITY · PI WITWER, KENNETH W · 2018 to 2022
$3.7M
Novel Approaches to Capture, Sorting, and Characterization of CNS-Origin Extracellular VesiclesR33MH118164 · NIMH · JOHNS HOPKINS UNIVERSITY · PI MACHAIRAKI, VASILIKI, WITWER, KENNETH W · 2020 to 2022
$2.2M
Novel Separation Methods for exRNA Carriers: Extracellular Vesicles, Lipoprotein Particles, and Protein AggregatesUH3CA241694 · NCI · JOHNS HOPKINS UNIVERSITY · PI WITWER, KENNETH W · 2021 to 2022
$1.1M
Targeted transcriptional activation of HIVR56AI147684 · NIAID · BECKMAN RESEARCH INSTITUTE/CITY OF HOPE · PI MORRIS, KEVIN V · 2020 to 2020
$440k
NCI NIH HHS UH3 CA241694NIAID NIH HHS R01 AI144997NIAID NIH HHS R56 AI147684NIDA NIH HHS R01 DA047807NIMH NIH HHS R33 MH118164
6 · The paper itself

Abstract

Extracellular vesicles (EVs) are esteemed as a promising delivery vehicle for various genetic therapeutics. They are relatively inert, non-immunogenic, biodegradable, and biocompatible. At least in rodents, they can even transit challenging bodily hurdles such as the blood-brain barrier. Constitutively shed by all cells and with the potential to interact specifically with neighboring and distant targets, EVs can be engineered to carry and deliver therapeutic molecules such as proteins and RNAs. EVs are thus emerging as an elegant in vivo gene therapy vector. Deeper understanding of basic EV biology-including cellular production, EV loading, systemic distribution, and cell delivery-is still needed for effective harnessing of these endogenous cellular nanoparticles as next-generation nanodelivery tools. However, even a perfect EV product will be challenging to produce at clinical scale. In this regard, we propose that vector transduction technologies can be used to convert cells either ex vivo or directly in vivo into EV factories for stable, safe modulation of gene expression and function. Here, we extrapolate from the current EV state of the art to a bright potential future using EVs to treat genetic diseases that are refractory to current therapeutics.

Indexed as

Extracellular VesiclesNanoparticlesGenetic TherapyProteinsRNAProteinsRNAcell targetingectosomesexosomesextracellular vesiclesnanoparticlenon-coding RNA

Identifiers

PMID36698310
PMCPMC10188631

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.