1. The 10th oligonucleotide therapy approved: golodirsen for Duchenne muscular dystrophy;Aartsma-Rus;Nucleic Acid Ther.,2020
2. Therapeutic antisense oligonucleotides are coming of age;Bennett;Annu. Rev. Med.,2019
3. Inotersen treatment for patients with hereditary transthyretin amyloidosis;Benson;N. Engl. J. Med.,2018
4. CFTR1 (Cystic Fibrosis Mutation Database) (2022). CFMDB Statistics. Retrieved October 22, 2022, from http://www.genet.sickkids.on.ca/StatisticsPage.html
5. CFTR2 (Clinical and Functional TRanslation of CFTR) (2022). CFTR2 Variant List History. Retrieved April 29, 2022, from https://cftr2.org/sites/default/files/CFTR2_29April2022.xlsx