Evidence map›Paper›PMID 33669654›Full record

ReviewPharmaceutics2021

Nanomedicine for Gene Delivery and Drug Repurposing in the Treatment of Muscular Dystrophies.

Ilaria Andreana, Mathieu Repellin, Flavia Carton, David Kryza, Stéphanie Briançon, Bénédicte Chazaud, Rémi Mounier, Silvia Arpicco, Manuela Malatesta, Barbara Stella and 1 more

Open access · goldAbstract readReview
In one paragraph

Review in Pharmaceutics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
1.6field-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

20 citing papers in PubMed, 27 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

11 authors at 3 institutions in 2 countries.

Ilaria AndreanaLaboratoire d'Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France.
Mathieu RepellinLaboratoire d'Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France.ORCID 0000-0003-0613-3667
Flavia CartonDepartment of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, Italy.
David KryzaLaboratoire d'Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France.
Stéphanie BriançonLaboratoire d'Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France.ORCID 0000-0001-7997-0410
Bénédicte ChazaudInstitut NeuroMyoGène, University of Lyon, INSERM U1217, CNRS UMR 5310, 8 Avenue Rockefeller, 69008 Lyon, France.ORCID 0000-0002-1262-502X
Rémi MounierInstitut NeuroMyoGène, University of Lyon, INSERM U1217, CNRS UMR 5310, 8 Avenue Rockefeller, 69008 Lyon, France.
Silvia ArpiccoDepartment of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, Italy.
Manuela MalatestaDepartment of Neurosciences, Biomedicine and Movement Sciences, Anatomy and Histology Section, University of Verona, Strada Le Grazie 8, 37134 Verona, Italy.ORCID 0000-0001-8196-9232
Barbara StellaDepartment of Drug Science and Technology, University of Turin, Via P. Giuria 9, 10125 Torino, Italy.ORCID 0000-0001-8266-6604
Giovanna LolloLaboratoire d'Automatique, de Génie des Procédés et de Génie Pharmaceutique, Université Claude Bernard Lyon 1, CNRS UMR 5007, 43 bd 11 Novembre 1918, 69622 Villeurbanne, France.
Université Claude Bernard Lyon 1 · FRUniversity of Turin · ITUniversity of Verona · IT

Funding

Università degli Studi di Verona 754345
6 · The paper itself

Abstract

Muscular Dystrophies (MDs) are a group of rare inherited genetic muscular pathologies encompassing a variety of clinical phenotypes, gene mutations and mechanisms of disease. MDs undergo progressive skeletal muscle degeneration causing severe health problems that lead to poor life quality, disability and premature death. There are no available therapies to counteract the causes of these diseases and conventional treatments are administered only to mitigate symptoms. Recent understanding on the pathogenetic mechanisms allowed the development of novel therapeutic strategies based on gene therapy, genome editing CRISPR/Cas9 and drug repurposing approaches. Despite the therapeutic potential of these treatments, once the actives are administered, their instability, susceptibility to degradation and toxicity limit their applications. In this frame, the design of delivery strategies based on nanomedicines holds great promise for MD treatments. This review focuses on nanomedicine approaches able to encapsulate therapeutic agents such as small chemical molecules and oligonucleotides to target the most common MDs such as Duchenne Muscular Dystrophy and the Myotonic Dystrophies. The challenge related to in vitro and in vivo testing of nanosystems in appropriate animal models is also addressed. Finally, the most promising nanomedicine-based strategies are highlighted and a critical view in future developments of nanomedicine for neuromuscular diseases is provided.

Indexed as

antisense oligonucleotidesCRISPR/Cas9Duchenne Muscular Dystrophymyotonic dystrophynanoparticlessmall molecules

Identifiers

PMID33669654
PMCPMC7922331
OpenAlexW3132721544

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.