Evidence map›Paper›PMID 37724045›Full record

ArticleCellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology2023

Tyrosine-Mutant AAV8 Vector Mediated Efficient and Safe Gene Transfer of Pigment Epithelium-Derived Factor to Mouse Lungs.

Débora P Ferreira, Sabrina V Martini, Helena A Oliveira, Adriana L Silva, Siddharth Shenoy, Daiqin Chen, Valentina Simon, Eric Han, Natalie E West, Jung Soo Suk and 4 more

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In one paragraph

Article in Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Review
  2. Article
  3. 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

14 authors.

Débora P FerreiraLaboratory of Cellular and Molecular Physiology, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Sabrina V MartiniLaboratory of Cellular and Molecular Physiology, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Helena A OliveiraLaboratory of Immunopharmacology, Oswaldo Cruz Institute, Fiocruz, Rio de Janeiro, Brazil.
Adriana L SilvaLaboratory of Cellular and Molecular Physiology, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Siddharth ShenoyCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Daiqin ChenCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Valentina SimonCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Eric HanCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Natalie E WestDepartment of Medicine, Johns Hopkins University, Baltimore, MD, USA.
Jung Soo SukCenter for Nanomedicine, Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Patricia R M RoccoLaboratory of Pulmonary Investigation, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Hilda Petrs-SilvaLaboratory of Gene Therapy and Viral Vectors, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Marcelo M MoralesLaboratory of Cellular and Molecular Physiology, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Fernanda F CruzLaboratory of Pulmonary Investigation, Carlos Chagas Filho Institute of Biophysics, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil, ffcruz@biof.ufrj.br.

Funding

New Inhalation Therapy for COPDR01HL136617 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI NEPTUNE, ENID R, SUK, JUNG SOO · 2017 to 2020
$1.6M
Brazilian Council for Scientific and Technological Development (CNPq)Coordination fort he Improvement of Higher-Level Personnel (CAPES)Cysic Fibrosis Foundation SUK18I0National Institute of Health R01HL136617National Institute of Science and Technology for Regenerative Medicine (INCT-REGENERA-CNPq)NHLBI NIH HHS R01 HL136617Rio de Janeiro State Research Supporting Foundation (FAPERJ)
6 · The paper itself

Abstract

BACKGROUND/

aimsRecombinant adeno-associated viruses (rAAV) are an important tool for lung targeted gene therapy. Substitution of tyrosine with phenylalanine residues (Y-F) in the capsid have been shown to protect the AAV vector from ubiquitin/proteasome degradation, increasing transduction efficiency. We tested the mutant Y733F-AAV8 vector for mucus diffusion, as well as the safety and efficacy of pigment epithelium-derived factor (PEDF) gene transfer to the lung.

methodsFor this purpose, Y733F-AAV8-PEDF (10

resultsThe tyrosine-mutant AAV8 vector was efficient at penetrating mucus in ex vivo assays and at transferring the gene to lung cells after in vivo instillation. Increased levels of transgene mRNA were observed 28 days after vector administration. Overexpression of PEDF did not affect in vivo lung parameters.

conclusionThese findings provide a basis for further development of Y733F-AAV8-based gene therapies for safe and effective delivery of PEDF, which has anti-angiogenic, anti-inflammatory and anti-fibrotic activities and might be a promising therapy for lung inflammatory disorders.

Indexed as

Eye ProteinsGene Transfer TechniquesSerpinsAnimalsMiceMice, Inbred C57BLNerve Growth FactorsPigment Epithelium-Derived FactorEye ProteinsNerve Growth FactorsPigment Epithelium-Derived FactorSerpinsGene therapy; Recombinant adeno-associated viruses serotype 8 (AAV8); Tyrosine mutant; Pigment epithelium-derived factor (PEDF)

Identifiers

PMID37724045
PMCPMC13156840

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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.