New Therapeutic Approaches for Familial Amyloidotic Polyneuropathy (FAP)
作者： Yukio Ando , Masaaki Nakamura , Mistuharu Ueda , Hirofumi Jono
DOI: 10.1007/978-3-642-00646-3_14
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摘要: Although liver transplantation is the only therapy to halt clinical manifestations of transthyretin (TTR) related familial amyloidotic polyneuropathy (FAP), has given rise several problems. An alternative treatment needed. After precursor protein amyloid fibrils in FAP was established 1983, different approaches have been investigated as an essential for FAP: (1) reduction variant TTR levels plasma, (2) down regulation gene mRNA, (3) inhibition deposition, (4) stabilization tetrameric structure and (5) replacement with normal (which can be achieved by or therapy). In this chapter, we introduce these research strategies trials discuss possibility optimal FAP.
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