TY  - JOUR
AB  - 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-specific molecular therapy in a clinical setting of FAP. The two therapeutic oligonucleotides bind to RNA in a sequence specific manner but exploit different mechanisms. Here we describe major developments that have led to the advent of therapeutic oligonucleotides for treatment of TTR-related disease.
AU  - Niemietz, Christoph
AU  - Chandhok, Gursimran
AU  - Chandhok, Gursimran Kaur
AU  - Schmidt, Hartmut
DA  - 2015-09-30
DO  - 10.3390/molecules201017944
KW  - transthyretin
KW  - familial amyloid polyneuropathy
KW  - antisense oligonucleotide
KW  - small-interfering RNA
KW  - liver
LA  - eng
N1  - Molecules 20 (2015) 10, 17944-17975
N1  - Finanziert durch den Open-Access-Publikationsfonds 2015/2016 der Westfälischen Wilhelms-Universität Münster (WWU Münster).
PY  - 2015-09-30
SN  - 1420-3049
TI  - Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
UR  - https://nbn-resolving.org/urn:nbn:de:hbz:6-47269568676
Y2  - 2024-11-22T01:21:34
ER  -