Concepedia

Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation

Yifat S. Oren, Michal Irony‐Tur Sinai, Anita Golec, Ofra Barchad-Avitzur, Venkateshwar Mutyam, Li Yao, Jeong S. Hong, E. Ozeri-Galai, Aurélie Hatton, Chen Leibson,

Journal of Cystic Fibrosis · 2021 · 52 citations · 35 references

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References

35

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Peter G. Middleton, Marcus Mall, Pavel Dřevı́nek et al. · New England Journal of Medicine · 2019 · 2.1K citations · Full text

, , +16

ESEfinder: a web resource to identify exonic splicing enhancers

Luca Cartegni · Nucleic Acids Research · 2003 · 1.6K citations · Full text

, , +14

Cystic Fibrosis

Steven M. Rowe, Stacey Miller, Eric J. Sorscher · New England Journal of Medicine · 2005 · 1.4K citations

, , +13

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

Harry Heijerman, Edward F. McKone, D.G. Downey et al. · The Lancet · 2019 · 1.4K citations · Full text

, , +14

Mutations in the Cystic Fibrosis Gene in Patients with Congenital Absence of the Vas Deferens

Miguel Chillón, Teresa Casals, Bernard Mercier et al. · New England Journal of Medicine · 1995 · 967 citations · Full text

, , +11