التفاصيل البيبلوغرافية
| العنوان: |
RNA interference in late-stage hereditary transthyretin amyloidosis: a clinicopathological study. |
| المؤلفون: |
Beck, Goichi, Yonenobu, Yuki, Kawai, Makiko, Ikenaka, Kensuke, Sera, Fusako, Ozono, Tatsuhiko, Shimada, Yuki, Yamashita, Rika, Sakata, Yasushi, Morii, Eiichi, Yoshinaga, Tsuneaki, Ando, Yukio, Murayama, Shigeo, Mochizuki, Hideki |
| المصدر: |
Journal of Neurology; Sep2023, Vol. 270 Issue 9, p4544-4548, 5p |
| مصطلحات موضوعية: |
CARDIAC amyloidosis, RNA interference, TRANSTHYRETIN, AMYLOIDOSIS, BRAIN natriuretic factor, AUTOPSY |
| مستخلص: |
Our findings lead us to strongly recommend that (i) genetic analysis should be performed in patients with polyneuropathy of unknown etiology, and (ii) that RNAi therapy should be started as early as possible in patients with genetically confirmed ATTRv amyloidosis. In contrast, excessive amyloid deposits were visible in the heart and sympathetic ganglia, and moderate amyloid deposits were found in several organs even after the periodic administration of patisiran for 21 months, likely because therapy was initiated at a late stage of the disease. Sural nerve biopsy showed no amyloid deposits in the endoneurium or perineurium, but a small amount of amyloid deposition was visible on the vessel wall by Congo red staining (Supplementary Figure S2). Although TTR synthesis in hepatocytes was significantly reduced, massive amyloid deposits were found in some organs, even after RNAi therapy, likely because of the late initiation of the therapy. [Extracted from the article] |
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| قاعدة البيانات: |
Complementary Index |
