In Vitro Studies to Evaluate the Intestinal Permeation of an Ursodeoxycholic Acid-Conjugated Oligonucleotide for Duchenne Muscular Dystrophy Treatment.

DMD myotubes Duchenne muscular dystrophy IEC-6 cells antisense oligonucleotides co-culture conjugated oligonucleotides exon skipping exosomes intestinal permeation ursodeoxycholic acid

Journal

Pharmaceutics
ISSN: 1999-4923
Titre abrégé: Pharmaceutics
Pays: Switzerland
ID NLM: 101534003

Informations de publication

Date de publication:
01 Aug 2024
Historique:
received: 04 07 2024
revised: 24 07 2024
accepted: 29 07 2024
medline: 31 8 2024
pubmed: 31 8 2024
entrez: 29 8 2024
Statut: epublish

Résumé

Delivery represents a major hurdle to the clinical advancement of oligonucleotide therapeutics for the treatment of disorders such as Duchenne muscular dystrophy (DMD). In this preliminary study, we explored the ability of 2'-

Identifiants

DOI: 10.3390/pharmaceutics16081023 PMID: 39204368
pubmed: 39204368
pii: pharmaceutics16081023
doi: 10.3390/pharmaceutics16081023
pii:
doi:

Types de publication

Journal Article

Langues

eng

Subventions

Organisme : University of Ferrara
ID : 2022-FAR.L-DA_018, A.D.; 2022-FAR.L-PB_001, B.P.; 2022-FAR.L-PD, D.P.; 2022-FAR.L-BM, M.B., 2021-FIRD M.B. and 2021-DOTT-DM-1061_FM_01_RIC, M.B

Auteurs

Marika Faiella (M)

Department of Translational Medicine, University of Ferrara, 44121 Ferrara, Italy.

Giada Botti (G)

Department of Chemical, Pharmaceutical and Agricultural Sciences, University of Ferrara, 44121 Ferrara, Italy.
Center for Translational Neurophysiology of Speech and Communication (CTNSC@UniFe), Italian Institute of Technology (IIT), 44121 Ferrara, Italy.
ORCID: 0000-0002-2942-1437

Alessandro Dalpiaz (A)

Department of Chemical, Pharmaceutical and Agricultural Sciences, University of Ferrara, 44121 Ferrara, Italy.
ORCID: 0000-0002-6648-2562

Lorenzo Gnudi (L)

Department of Environmental and Prevention Sciences, University of Ferrara, 44121 Ferrara, Italy.

Aurélie Goyenvalle (A)

University Paris-Saclay, UVSQ, Inserm, END-ICAP, 78000 Versailles, France.
ORCID: 0000-0003-3938-1165

Barbara Pavan (B)

Center for Translational Neurophysiology of Speech and Communication (CTNSC@UniFe), Italian Institute of Technology (IIT), 44121 Ferrara, Italy.
Department of Neuroscience and Rehabilitation-Section of Physiology, University of Ferrara, 44121 Ferrara, Italy.
ORCID: 0000-0001-8942-9310

Daniela Perrone (D)

Department of Environmental and Prevention Sciences, University of Ferrara, 44121 Ferrara, Italy.
ORCID: 0000-0001-5189-011X

Matteo Bovolenta (M)

Department of Translational Medicine, University of Ferrara, 44121 Ferrara, Italy.
ORCID: 0000-0002-9937-009X

Elena Marchesi (E)

Department of Chemical, Pharmaceutical and Agricultural Sciences, University of Ferrara, 44121 Ferrara, Italy.
ORCID: 0000-0002-5402-4732

Classifications MeSH