Evidence map›Paper›PMID 35456263›Full record

ArticleJournal of clinical medicine2022

Mesoporous Silica-Based Nanoparticles as Non-Viral Gene Delivery Platform for Treating Retinitis Pigmentosa.

Lourdes Valdés-Sánchez, Sara Borrego-González, Adoración Montero-Sánchez, Simone Massalini, Berta de la Cerda, Aránzazu Díaz-Cuenca, Francisco J Díaz-Corrales

Open access · goldAbstract read
In one paragraph

Article in Journal of clinical medicine, 2022. 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
1.7field-weighted citation impact, top 16% 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, 22 citations in OpenAlex.

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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

7 authors at 2 institutions in 1 country.

Lourdes Valdés-SánchezRegeneration and Cell Therapy Department, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Junta de Andalucía, CSIC, Universidad de Sevilla, Universidad Pablo de Olavide, 41092 Sevilla, Spain.ORCID 0000-0001-9385-3915
Sara Borrego-GonzálezMaterials Science Institute of Seville (ICMS), Joint CSIC-University of Seville Center, 41092 Seville, Spain.
Adoración Montero-SánchezRegeneration and Cell Therapy Department, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Junta de Andalucía, CSIC, Universidad de Sevilla, Universidad Pablo de Olavide, 41092 Sevilla, Spain.
Simone MassaliniRegeneration and Cell Therapy Department, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Junta de Andalucía, CSIC, Universidad de Sevilla, Universidad Pablo de Olavide, 41092 Sevilla, Spain.
Berta de la CerdaRegeneration and Cell Therapy Department, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Junta de Andalucía, CSIC, Universidad de Sevilla, Universidad Pablo de Olavide, 41092 Sevilla, Spain.ORCID 0000-0001-5603-6473
Aránzazu Díaz-CuencaMaterials Science Institute of Seville (ICMS), Joint CSIC-University of Seville Center, 41092 Seville, Spain.ORCID 0000-0002-5790-4452
Francisco J Díaz-CorralesRegeneration and Cell Therapy Department, Andalusian Molecular Biology and Regenerative Medicine Centre (CABIMER), Junta de Andalucía, CSIC, Universidad de Sevilla, Universidad Pablo de Olavide, 41092 Sevilla, Spain.
Centro Andaluz de Biología Molecular y Medicina Regenerativa · ESInstituto de Ciencia de Materiales de Sevilla · ES

Funding

Consejería de Salud, Igualdad y Políticas Sociales PI-0099-2018Instituto de Salud Carlos III CP15/00071Ministerio de Economía, Ciencia e Innovación P10-CTS-6681
6 · The paper itself

Abstract

backgroundGene therapy is a therapeutic possibility for retinitis pigmentosa (RP), in which therapeutic transgenes are currently delivered to the retina by adeno-associated viral vectors (AAVs). Although their safety and efficacy have been demonstrated in both clinical and preclinical settings, AAVs present some technical handicaps, such as limited cargo capacity and possible immunogenicity in repetitive doses. The development of alternative, non-viral delivery platforms like nanoparticles is of great interest to extend the application of gene therapy for RP.

methodsAmino-functionalized mesoporous silica-based nanoparticles (N-MSiNPs) were synthesized, physico-chemically characterized, and evaluated as gene delivery systems for human cells in vitro and for retinal cells in vivo. Transgene expression was evaluated by WB and immunofluorescence. The safety evaluation of mice subjected to subretinal injection was assessed by ophthalmological tests (electroretinogram, funduscopy, tomography, and optokinetic test).

resultsN-MSiNPs delivered transgenes to human cells in vitro and to retinal cells in vivo. No adverse effects were detected for the integrity of the retinal tissue or the visual function of treated eyes. N-MSiNPs were able to deliver a therapeutic transgene candidate for RP,

conclusionsN-MSiNPs are safe for retinal delivery and thus a potential alternative to viral vectors.

Indexed as

gene therapymesoporous silicananoparticlesPRPF31retinitis pigmentosa

Identifiers

PMID35456263
PMCPMC9026300
OpenAlexW4223956337

What OpenQuestion holds

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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.