The safety and toxicity profile of SPL84, an inhaled antisense oligonucleotide for treatment of cystic fibrosis patients with the 3849 +10kb C->T mutation, supports a Phase 1/2 clinical study

Friedman, Lital ; Avitzur, Ofra-Barchad ; Galai, Efrat Ozeri ; Ferrari, Nicolay ; Choen, Asa ; Dahan, Sara ; Mordechai, Tamar ; Hart, Gili

Taylor & Francis 2023

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