Abstract: Disclosed are conjugates of an oligonucleotide and a peptide covalently bonded or linked via a linker to the oligonucleotide, the peptide including at least one cationic domain comprising at least 4 amino acid residues and at least one hydrophobic domain comprising at least 3 amino acid residues, provided that the peptide includes a total of 7 to 40 amino acid residues and does not include any artificial amino acid residues; and the oligonucleotide including a total of 12 to 40 contiguous nucleobases, where at least 12 contiguous nucleobases are complementary to a target sequence in a human dystrophin gene.

Abstract: Disclosed are methods of treating a subject having Duchenne muscular dystrophy. The method includes administration of 1 mg/kg to 60 mg/kg of a conjugate of an oligonucleotide and a peptide covalently bonded or linked via a linker to the oligonucleotide to the subject (e.g., a subject amenable to exon 51 skipping). The peptide including at least one cationic domain including at least 4 amino acid residues and at least one hydrophobic domain including at least 3 amino acid residues, provided that the peptide includes a total of 7 to 40 amino acid residues, and provided that the at least one cationic domain includes a beta-alanine residue in combination with arginine and/or histidine residues. The oligonucleotide including a total of 12 to 40 contiguous nucleobases, wherein at least 12 contiguous nucleobases are complementary to a target sequence in a human dystrophin gene.

Abstract: Disclosed are methods of treating a subject having myotonic dystrophy type 1 (DM1).

Abstract: The present invention relates to linkers for connecting a carrier molecule to a therapeutic molecule to form a conjugate, in particular linkers formed of amino acids such as glutamic acid, succinic acid, and gamma-aminobutyric acid. The present invention further relates to a conjugate comprising a linker of the invention, and the use of said conjugate in the treatment of various diseases.

Abstract: The present invention relates to peptides, in particular cell-penetrating peptides, and to conjugates of such cell-penetrating peptides with a therapeutic molecule. The present invention further relates to use of such peptides or conjugates in methods of treatment or as a medicament, especially in the treatment of genetic disorders and in particular muscular dystrophies such as Duchenne muscular dystrophy.

Abstract: the present invention relates to peptides, in particular cell-penetrating peptides, having a first hydrophobic domain positioned at the C-terminus of the peptide and a second hydrophobic domain positioned at the N-terminus of the peptide, and to conjugates of such cell-penetrating peptides with a therapeutic molecule. The present invention further relates to use of such peptides or conjugates in methods of treatment or as a medicament, especially in the treatment of genetic disorders and in particular muscular dystrophies such as Duchenne muscular dystrophy.