Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of

Chitosan Gene Silencing Morpholinos / genetics Oligonucleotides, Antisense / genetics
DUX4 silencing FSHD PMO AON nanobubbles

Journal

Nucleic acid therapeutics
ISSN: 2159-3345
Titre abrégé: Nucleic Acid Ther
Pays: United States
ID NLM: 101562758

Informations de publication

Date de publication:
06 2021
Historique:
pubmed: 18 7 2020
medline: 14 1 2022
entrez: 18 7 2020
Statut: ppublish

Résumé

Orphan drugs, including antisense oligonucleotides (AONs), siRNAs/miRNAs, Cas9 nuclease, and recombinant genes, have recently been made available for rare diseases. However, the main bottleneck for these new therapies is delivery. Drugs/synthetic genes need to reach the affected tissues with minimal off-target effects and immune reactions. AON molecules are currently delivered as backboned naked compounds or via viral vectors. Nanocarriers are considered promising vehicles, able to improve drug distribution by organ targeting and limiting safety issues. We tested perfluoropentane-based nanobubbles (NBs) as vehicles for loading phosphorodiamidate morpholino (PMO) AON to suppress DUX4 expression in a facioscapulohumeral muscular dystrophy cell model.

Identifiants

DOI: 10.1089/nat.2020.0862 PMID: 32679000
pubmed: 32679000
doi: 10.1089/nat.2020.0862
doi:

Substances chimiques

Morpholinos 0
Oligonucleotides, Antisense 0
Chitosan 9012-76-4

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

201-207

Subventions

Organisme : Department of Health
Pays : United Kingdom

Auteurs

Maria Sofia Falzarano (MS)

Unit of Medical Genetics, University of Ferrara, Ferrara, Italy.

Monica Argenziano (M)

Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy.

Anne Chalotte Marsollier (AC)

NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom.

Virginie Mariot (V)

NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom.

Davide Rossi (D)

Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy.

Rita Selvatici (R)

Unit of Medical Genetics, University of Ferrara, Ferrara, Italy.

Julie Dumonceaux (J)

NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom.

Roberta Cavalli (R)

Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy.

Alessandra Ferlini (A)

Unit of Medical Genetics, University of Ferrara, Ferrara, Italy.
The Dubowitz Neuromuscular Centre, UCL Street Institute of Child Health, London, United Kingdom.

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Classifications MeSH

questionsmedicales.fr › Glucides › Polyosides › Chitine › Chitosane › Chitosan-Shelled Nanobubbles Irreversibly...
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Chitosan-Shelled Nanobubbles Irreversibly...
questionsmedicales.fr › Acides nucléiques, nucléotides et nucléosides › Nucléotides › Polynucléotides › Oligonucléotides › Morpholinos › Chitosan-Shelled Nanobubbles Irreversibly...
questionsmedicales.fr › Actions chimiques et utilisations › Utilisations spécialisées de produits chimiques › Produits chimiques de laboratoire › Sondes moléculaires › Sondes d'acide nucléique › Éléments antisens (génétique) › Oligonucléotides antisens › Chitosan-Shelled Nanobubbles Irreversibly...