Antisense oligonucleotides (ASOs) are synthetic single-stranded DNA molecules that hybridize with mRNA targets to degrade them or otherwise prevent their translation by cellular machinery. ASOs have ...
Purpose: To determine the comparative frequency of 93 CFTR mutations in U.S. individuals with a clinical diagnosis of cystic fibrosis (CF). Methods: A total of 5,840 CF chromosomes from Caucasians, ...
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