Orthotopic liver transplantation (OLT) that eliminates hepatic production of variant transthyretin (TTR) in patients with hereditary TTR amyloidosis is the only specific therapy that has been shown to be effective. Unfortunately, OLT does not stop progression of the disease in some patients since normal TTR can continue to be deposited as amyloid fibrils. In the present study, we have advanced in our attempts to develop a specific antisense oligonucleotide that will suppress hepatic production of TTR. With favorable response in TTR transgenic mice, studies have progressed to safety analysis in non-human primates.
ASJC Scopus subject areas
- Internal Medicine