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对PXE的新疗法:复合ENPP1酶疗法

Ida Joely Jacobs1, Dora Obiri-Yeboah1, Paul R Stabach2

  • 1Department of Biochemistry and Molecular Biology, Sidney Kimmel Medical College, Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, PA, USA; PXE International Center of Excellence in Research and Clinical Care, Thomas Jefferson University, Philadelphia, PA, USA.

Molecular therapy : the journal of the American Society of Gene Therapy
|September 29, 2024
PubMed
概括

一种新的治疗方法BL-1118对Pseudoxanthoma elasticum (PXE) 有望. 这种酶疗法有效地增加了血中铁酸盐 (PPi) 水平,并减少了与PXE有关的小鼠的宫外化.

关键词:
ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC6ABCC在 ENPP1 中使用.宫外化是一种非宫外化.酶疗法是一种酶疗法.伪桑托马弹性 (pseudoxanthoma elasticum) 是一种类型的酸铁酸盐的使用方法

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