fig5

RNA antisense and silencing strategies using synthetic drugs for rare muscular and neuromuscular diseases

Figure 5. Different types of ASOs developed at the preclinical and clinical stages. Squares, circles, and assorted colors represent the diverse types of nucleotides displayed in Figure 4. The linkage between each nucleotide might also vary, for instance, the phosphorothioate Sp and Rp diastereoisomers. In a gapmer, the yellow circles represent non-modified nucleotides allowing cleavage by RNase H of the annealed complementary mRNA.

Rare Disease and Orphan Drugs Journal
ISSN 2771-2893 (Online)
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