Article
Targeted suppression of an amyloidogenic transthyretin with antisense oligonucleotides.
Muscle & nerve - 1 May 2006
Benson Merrill D, Kluve-Beckerman Barbara, Zeldenrust Steven R, Siesky Angela M, Bodenmiller Diane M, Showalter Aaron D, Sloop Kyle W
Abstract excerpt
Transthyretin (TTR) amyloidosis, the most common form of hereditary systemic amyloidosis, is characterized clinically by adult-onset axonal neuropathy and restrictive cardiomyopathy. More than 85 mutations in transthyretin have been found to cause this hereditary disease. Since essentially all circulating TTR is of hepatic origin, orthotopic liver transplantation has been used as the only specific form of...
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