×

Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy

  • US 10,563,198 B2
  • Filed: 03/11/2016
  • Issued: 02/18/2020
  • Est. Priority Date: 03/11/2015
  • Status: Active Grant
First Claim
Patent Images

1. An antisense oligoribonucleotide capable of preventing or reducing exon 73 inclusion into a human COL7A1 mRNA when the mRNA is produced by splicing from a pre-mRNA in a human cell, wherein the oligoribonucleotide comprises a nucleotide sequence selected from SEQ ID NO:

  • 5, 24, 25, 26, 27, 28, 39, 40, 41, 42, 43, 29 and 35, the oligoribonucleotide has a length of between 16 and 24 nucleotides, and the oligoribonucleotide comprises at least one of (i) a nucleotide residue having a modified base, (ii) a modified backbone, and (iii) a non-natural internucleoside linkage.

View all claims
  • 2 Assignments
Timeline View
Assignment View
    ×
    ×