Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene.

Venturini, Arianna; Borrelli, Anna; Musante, Ilaria; Scudieri, Paolo; Capurro, Valeria; Renda, Mario; Pedemonte, Nicoletta; Galietta, Luis J V

Venturini, Arianna; Borrelli, Anna; Musante, Ilaria; Scudieri, Paolo; Capurro, Valeria; Renda, Mario; Pedemonte, Nicoletta; Galietta, Luis J V.

Affiliation

Venturini A; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
Borrelli A; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
Musante I; U.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Scudieri P; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), Università degli Studi di Genova, 16132 Genova, Italy.
Capurro V; U.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Renda M; Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (DINOGMI), Università degli Studi di Genova, 16132 Genova, Italy.
Pedemonte N; U.O.C. Genetica Medica, IRCCS Istituto Giannina Gaslini, 16147 Genova, Italy.
Galietta LJV; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.

Int J Mol Sci ; 22(21)2021 Nov 04.

Article de En | MEDLINE | ID: mdl-34769402

Sujet(s)
Mots clés

CFTR rescue; chloride channel; cystic fibrosis; nonsense mutation; readthrough therapy

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Texte intégral: 1 Collection: 01-internacional Base de données: MEDLINE Sujet principal: Pyrazoles / Pyridines / Pyrrolidines / Codon non-sens / Protéine CFTR / Mucoviscidose / Benzodioxoles / Dégradation des ARNm non-sens / Aminopyridines Limites: Humans Langue: En Journal: Int J Mol Sci Année: 2021 Type de document: Article Pays d'affiliation: Italie

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