Evidence map›Paper›PMID 28361998›Full record

ArticleScientific reports2017

Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection.

Sarah J Wassmer, Livia S Carvalho, Bence György, Luk H Vandenberghe, Casey A Maguire

Abstract read
In one paragraph

Article in Scientific reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 98 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
98citing 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

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

  1. Pooled it
  2. Article
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  5. Review
  6. Review
  7. Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  8. Round window membrane extracellular vesicles facilitate inner ear drug delivery.Journal of controlled release : official journal of the Controlled Release Society · 2025
    Article
  9. Review
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  11. Review
  12. Review
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  15. Article
  16. Article
  17. Article
  18. Article
  19. Review
  20. Inhibition of Melanin Synthesis and Inflammation by Exosomes Derived fromJournal of microbiology and biotechnology · 2025
    Article

38 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

5 authors.

Sarah J WassmerHarvard Stem Cell Institute, Harvard University, Cambridge, MA, USA.
Livia S CarvalhoHarvard Stem Cell Institute, Harvard University, Cambridge, MA, USA.
Bence GyörgyDepartment of Neurobiology and Howard Hughes Medical Institute, Harvard Medical School, 220 Longwood Avenue, Boston, 02115 MA, USA.
Luk H VandenbergheHarvard Stem Cell Institute, Harvard University, Cambridge, MA, USA.
Casey A MaguireOcular Genomics Institute, Department of Ophthalmology, Harvard Medical School, Boston, MA, USA.

Funding

Interdepartmental Neuroscience CenterP30NS045776 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BREAKEFIELD, XANDRA OWENS, TANNOUS, BAKHOS A · 2003 to 2018
$11.1M
Broad Spectrum Molecular Therapy for Blinding Retina DisordersDP1EY023177 · NEI · UNIVERSITY OF PENNSYLVANIA · PI BENNETT, JEAN · 2012 to 2015
$3.2M
NEI NIH HHS DP1 EY023177NINDS NIH HHS P30 NS045776
6 · The paper itself

Abstract

Widespread gene transfer to the retina is challenging as it requires vector systems to overcome physical and biochemical barriers to enter and diffuse throughout retinal tissue. We investigated whether exosome-associated adeno-associated virus, (exo-AAV) enabled broad retinal targeting following intravitreal (IVT) injection, as exosomes have been shown to traverse biological barriers and mediate widespread distribution upon systemic injection. We packaged an AAV genome encoding green fluorescent protein (GFP) into conventional AAV2 and exo-AAV2 vectors. Vectors were IVT injected into the eyes of adult mice. GFP expression was noninvasively monitored by fundus imaging and retinal expression was analyzed 4 weeks post-injection by qRT-PCR and histology. Exo-AAV2 outperformed conventional AAV2 in GFP expression based on fundus image analysis and qRT-PCR. Exo-AAV2 demonstrated deeper penetration in the retina, efficiently reaching the inner nuclear and outer plexiform, and to a lesser extent the outer nuclear layer. Cell targets were ganglion cells, bipolar cells, Müller cells, and photoreceptors. Exo-AAV2 serves as a robust gene delivery tool for murine retina, and the simplicity of production and isolation should make it widely applicable to basic research of the eye.

Indexed as

AnimalsDependovirusExosomesGenetic VectorsGene Transfer TechniquesGreen Fluorescent ProteinsIntravitreal InjectionsMaleMiceRetinaGreen Fluorescent Proteins

Identifiers

PMID28361998
PMCPMC5374486

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.