Evidence map›Paper›PMID 26359319›Full record

ArticleHuman gene therapy2015

Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection.

Daniel M Lipinski, Chris A Reid, Sanford L Boye, James J Peterson, Xiaoping Qi, Shannon E Boye, Michael E Boulton, William W Hauswirth

Open access · greenAbstract read
In one paragraph

Article in Human gene therapy, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
0.5field-weighted citation impact, top 26% 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

10 citing papers in PubMed, 25 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Annexin A8 deficiency delays atherosclerosis progression.Clinical and translational medicine · 2025
    Article
  5. A Simple Model to Study Mosaic Gene Expression in 3D Endothelial Spheroids.Journal of cardiovascular development and disease · 2024
    Article
  6. Article
  7. Article
  8. Article
  9. Article
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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

8 authors at 2 institutions in 2 countries.

Daniel M Lipinski1 Department of Ophthalmology, College of Medicine, University of Florida , Gainesville, Florida .
Chris A Reid1 Department of Ophthalmology, College of Medicine, University of Florida , Gainesville, Florida .
Sanford L Boye1 Department of Ophthalmology, College of Medicine, University of Florida , Gainesville, Florida .
James J Peterson1 Department of Ophthalmology, College of Medicine, University of Florida , Gainesville, Florida .
Xiaoping Qi3 Department of Ophthalmology, Indiana University School of Medicine, Indiana University , Indianapolis, Indiana.
Shannon E Boye1 Department of Ophthalmology, College of Medicine, University of Florida , Gainesville, Florida .
Michael E Boulton3 Department of Ophthalmology, Indiana University School of Medicine, Indiana University , Indianapolis, Indiana.
William W Hauswirth1 Department of Ophthalmology, College of Medicine, University of Florida , Gainesville, Florida .
University of Florida · USIndiana University – Purdue University Indianapolis · US

Funding

Engineering AAV for safe and efficient gene delivery to the human retinaR01EY024280 · NEI · UNIVERSITY OF FLORIDA · PI Shannon Elizabeth Boye · 2014 to 2026
$7.5M
Vision Research CoreP30EY021721 · NEI · UNIVERSITY OF FLORIDA · PI HAUSWIRTH, WILLIAM W · 2011 to 2015
$2.8M
Targeting gamma-secretase activation for anti-angiogenesisR01EY018358 · NEI · UNIVERSITY OF TEXAS MED BR GALVESTON · PI BOULTON, MICHAEL EDWIN · 2007 to 2012
$2.2M
NEI NIH HHS EY018358NEI NIH HHS P30EY021721NEI NIH HHS R01 EY024280
6 · The paper itself

Abstract

The ability to effectively deliver genetic material to vascular endothelial cells remains one of the greatest unmet challenges facing the development of gene therapies to prevent diseases with underlying vascular etiology, such as diabetes, atherosclerosis, and age-related macular degeneration. Herein, we assess the effectiveness of an rAAV2-based capsid mutant vector (Y272F, Y444F, Y500F, Y730F, T491V; termed QuadYF+TV) with strong endothelial cell tropism at transducing the vasculature after systemic administration. Intravenous injection of QuadYF+TV resulted in widespread transduction throughout the vasculature of several major organ systems, as assessed by in vivo bioluminescence imaging and postmortem histology. Robust transduction of lung tissue was observed in QuadYF+TV-injected mice, indicating a role for intravenous gene delivery in the treatment of chronic diseases presenting with pulmonary complications, such as α1-antitrypsin deficiency. The QuadYF+TV vector cross-reacted strongly with AAV2 neutralizing antibodies, however, indicating that a targeted delivery strategy may be required to maximize clinical translatability.

Indexed as

Administration, IntravenousGenetic TherapyTransduction, GeneticAnimalsCapsid ProteinsDependovirusEndothelial CellsGenetic VectorsMiceMice, Inbred BALB CMice, Inbred C57BLMutationVascular DiseasesCapsid Proteins

Identifiers

PMID26359319
PMCPMC4651034
OpenAlexW2132752221

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.