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Publikováno v:
Molecules
Molecules, Vol 20, Iss 10, Pp 17944-17975 (2015)
Molecules, Vol 20, Iss 10, Pp 17944-17975 (2015)
The liver has become an increasingly interesting target for oligonucleotide therapy. Mutations of the gene encoding transthyretin (TTR), expressed in vast amounts by the liver, result in a complex degenerative disease, termed familial amyloid polyneu