Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Eric W.F.W. Alton, David K Armstrong, Deborah Ashby et al. · The Lancet Respiratory Medicine · 2015 · 424 citations · Full text

Pharmacy, Molecular Medicine, Pharmacotherapy +17

Nonsense-mediated mRNA decay affects nonsense transcript levels and governs response of cystic fibrosis patients to gentamicin

Liat Linde, Stephanie Boelz, Malka Nissim‐Rafinia et al. · Journal of Clinical Investigation · 2007 · 294 citations · Full text

Life Sciences, Genetics, Molecular Biology +17

Association of a nonsense mutation (W1282X), the most common mutation in the Ashkenazi Jewish cystic fibrosis patients in Israel, with presentation of severe disease.

Tzipora Shoshani, Arie Augarten, Ephraim Gazit et al. · PubMed · 1992 · 202 citations

Life Sciences, Genetics, Molecular Genetics +12

Construction of a functional human suppressor tRNA gene: an approach to gene therapy for β-thalassaemia

Gary F. Temple, Andrée M. Dozy, Kenneth L. Roy et al. · Nature · 1982 · 168 citations

Life Sciences, Genetics, Molecular Genetics +7

Engineered transfer RNAs for suppression of premature termination codons

John D. Lueck, Jae Seok Yoon, Alfredo Perales‐Puchalt et al. · Nature Communications · 2019 · 151 citations · Full text

Life Sciences, Genetics, Molecular Biology +17