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Updated: Jul 12, 2025

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
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遗传性跨甲基氨基酸神经病变:病理生理学,生物标志物和治疗方面的进展

David Adams1, Yoshiki Sekijima2, Isabel Conceição3

  • 1Department of Neurology, Bicêtre Centre Hospitalo Universitaire, AP-HP, INSERM U 1195, University Paris Saclay, Le Kremlin Bicetre, France.

The Lancet. Neurology
|October 20, 2023
PubMed
概括

遗传性转基因粉样性多神经病变 (ATTR) 是一种严重的遗传性疾病. 像小干扰RNAs (siRNAs) 这样的新疗法在停止神经病变方面表现有前途,但中枢神经系统的影响仍然是一个挑战.

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