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Updated: Sep 10, 2025

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

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グライコゲン貯蔵症タイプIaにおける代謝治療としての肝臓移植

Deniz Turkum Atikcan Simsek1, Elif Melek Avcı Dursun2, Merve Senay Ozcalik1

  • 1Department of Endocrinology and Metabolism, Baskent University, Cankaya, Ankara 06530, Turkey.

JCEM case reports
|August 21, 2025
PubMed
まとめ

肝臓移植は,代謝問題を解消し,骨の健康を改善し,グリコゲン貯蔵病タイプIa (GSD Ia) の最終的な治療法を提供します. この治療は,従来の治療に反応しないGSD Iaの患者にとって極めて重要です.

キーワード:
グライコゲン貯蔵病型Ia肝臓腺腫肝臓移植代謝障害骨粗鬆症

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