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Source: Molecules
Condition: Amyloidosis
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Molecules, Vol. 20, Pages 17944-17975: Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
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 polyneuropathy (FAP). Misfolded variants of TTR are linked to the establishment of extracellular protein deposition in various tissues, including the heart and the peripheral nervous system. Recent progress in the chemistry and formulation of antisense (ASO) and small interfering RNA (siRNA) designed for a knockdown of TTR mRNA in the liver has allowed to address the issue of gene-spe...
Source: Molecules - September 30, 2015 Category: Chemistry Authors: Christoph NiemietzGursimran ChandhokHartmut Schmidt Tags: Review Source Type: research
