Antisense oligonucleotide-mediated exon skipping as systemic therapy for recessive dystrophic epidermolysis bullosa (rdeb).

Pasmooij, A. M. G., van den Akker, P. C., Bremer, J. & Jonkman, M. F., 11-May-2017, Patent No. WO2017078526, Priority date 5-Nov-2015

Research output: PatentProfessional

The invention relates to the field of medicine, in particular to means and methods for the treatment of the devastating skin blistering disease Recessive dystrophic epidermolysis bullosa (RDEB). Provided is an isolated antisense oligonucleotide (AON) of 14 to 35 nucleotides in length capable of binding to a target sequence of exon 105 in the COL7A1 gene, said target sequence comprising a stretch of at least 17 contiguous nucleotides of the exon 105 sequence and comprising at least one of the nucleotide sequences 5'-CCUGGUA-3' and 5'-AGGAG-3'.
Original languageEnglish
Patent numberWO2017078526
Priority date05/11/2015
Publication statusPublished - 11-May-2017

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