Abstract: Provided are peptide nucleic acid derivatives targeting a 3? splice site of the human tyrosinase pre-mRNA. The peptide nucleic acid derivatives potently induce a splice variant of the human tyrosinase mRNA in cells, and are useful to safely treat dermatological indications or conditions involving the human tyrosinase protein upon topical administration.

Abstract: Provided are peptide nucleic acid derivatives targeting a part of the human HIF-1? pre-mRNA. The peptide nucleic acid derivatives potently induce exon skipping to yield splice variants of HIF-1? mRNA in cells, and are useful to treat indications or conditions involving the overexpression of HIF-1?.

Abstract: Provided are peptide nucleic acid derivatives targeting a 3? splice site of the human SNAP25 pre-mRNA. The peptide nucleic acid derivatives potently induce at least a splice variant of the human SNAP25 mRNA in cells, and are useful to safely treat dermatological indications or conditions involving the expression of the human SNAP25 protein by topical administration.

Abstract: The current invention provides peptide nucleic acid derivatives targeting the 3? splice site of exon 4 in the human SCN9A pre-mRNA. The peptide nucleic acid derivatives potently induce SCN9A mRNA splice variant(s) lacking the SCN9A exon 4 in cells, and are useful to safely treat pains or conditions involving Nav1.7 activity.

Abstract: Provided are peptide nucleic acid derivatives targeting a part of the human HIF-1? pre-mRNA. The peptide nucleic acid derivatives potently induce exon skipping to yield splice variants of HIF-1? mRNA in cells, and are useful to treat indications or conditions involving the overexpression of HIF-1?.

Abstract: Provided are peptide nucleic acid derivatives targeting the 5? splice site of “exon 5” within the human androgen receptor pre-mRNA. The peptide nucleic acid derivatives potently induce splice variants of the androgen receptor mRNA in cells, and are useful to safely treat dermatological indications or conditions involving androgenic activity upon topical administration.

Abstract: The current invention provides peptide nucleic acid derivatives targeting the 3? splice site of exon 4 in the human SCN9A pre-mRNA. The peptide nucleic acid derivatives potently induce SCN9A mRNA splice variant(s) lacking the SCN9A exon 4 in cells, and are useful to safely treat pains or conditions involving Nav1.7 activity.

Abstract: Provided are peptide nucleic acid derivatives targeting a 3? splice site of the human SNAP25 pre-mRNA. The peptide nucleic acid derivatives potently induce at least a splice variant of the human SNAP25 mRNA in cells, and are useful to safely treat dermatological indications or conditions involving the expression of the human SNAP25 protein by topical administration.

Abstract: A peptide nucleic acid derivative of Formula I is provided to tightly bind to a splice site within a pre-mRNA in a sequence specific manner. Given with excellent cell membrane permeability and strong affinity for RNA, the said peptide nucleic acid derivative induces exon skipping in cells treated with the peptide nucleic acid at sub-femtomolar concentration as “naked” oligonucleotide. The said compound shows therapeutic activity in subjects upon systemic administration even at 1 ?g/Kg or less, and therefore is useful to treat a disease or symptom at affordable treatment cost.

Abstract: The current invention provides peptide nucleic acid derivatives targeting a part of the human SCN9A pre-mRNA. The peptide nucleic acid derivatives potently induce splice variants of the SCN9A mRNA in cells, and are useful to safely treat pains or conditions involving Nav1.7 activity.