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. Author manuscript; available in PMC: 2021 Apr 1.

Published in final edited form as: Expert Opin Drug Discov. 2020 Jan 30;15(4):443–456. doi: 10.1080/17460441.2020.1718100

Figure 3. — A, Exon 44 deletion can be treated with (i) exon 45 skipping using antisense oligonucleotides, (ii) exon 45 deletion using CRISPR, (iii) exon 45 re-framing using CRISPR, and (iv) exon 45 skipping using CRISPR. B, Exon 2 duplication can be treated with (i) skipping one copy of exon 2 using antisense oligonucleotides, (ii) skipping both copies of exon 2 using antisense oligonucleotides to induce IRES-mediated translation of a N-terminal partially truncated dystrophin, and (iii) deleting one copy of exon 2 using CRISPR. AON, antisense oligonucleotides; gRNA, guide RNA; IRES, internal ribosome entry site.

Figure 3. — A, Exon 44 deletion can be treated with (i) exon 45 skipping using antisense oligonucleotides, (ii) exon 45 deletion using CRISPR, (iii) exon 45 re-framing using CRISPR, and (iv) exon 45 skipping using CRISPR. B, Exon 2 duplication can be treated with (i) skipping one copy of exon 2 using antisense oligonucleotides, (ii) skipping both copies of exon 2 using antisense oligonucleotides to induce IRES-mediated translation of a N-terminal partially truncated dystrophin, and (iii) deleting one copy of exon 2 using CRISPR. AON, antisense oligonucleotides; gRNA, guide RNA; IRES, internal ribosome entry site.