TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients

Abstract excerpt

OBJECTIVE: Patients with hereditary transthyretin (TTR) amyloidosis (hATTR) often experience disease progression after orthotopic liver transplant (POLT) due in part to wild type ATTR amyloid deposition. The management strategy is not defined. We propose that TTR gene silencing with an antisense oligonucleotide or a small interfering ribonucleic acid may be a treatment for these patients. METHODS: We reviewed the...

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