Targeted conversion of the transthyretin gene in vitro and in vivo

  title={Targeted conversion of the transthyretin gene in vitro and in vivo},
  author={Masahiko Nakamura and Yukio Ando and Shunji Nagahara and Akiko Sano and Takahiro Ochiya and Shuichiro Maeda and Takahiro Kawaji and Masahiro Ogawa and Aiko Hirata and Hisayasu Terazaki and Katsuki Haraoka and Hidenobu Tanihara and Masumi Ueda and Makoto Uchino and Ken-ichi Yamamura},
  journal={Gene Therapy},
Familial amyloidotic polyneuropathy (FAP) is the common form of hereditary generalized amyloidosis and is characterized by the accumulation of amyloid fibrils in the peripheral nerves and other organs. Liver transplantation has been utilized as a therapy for FAP, because the variant transthyretin (TTR) is predominantly synthesized by the liver, but this therapy is associated with several problems. Thus, we need to develop a new treatment that prevents the production of the variant TTR in the… CONTINUE READING
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