کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
8513400 1556495 2018 7 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Lipidic Nanoparticles Comprising Phosphatidylinositol Mitigate Immunogenicity and Improve Efficacy of Recombinant Human Acid Alpha-Glucosidase in a Murine Model of Pompe Disease
ترجمه فارسی عنوان
نانوذرات لیپیدی شامل فسفاتیدیلینوزیتول موجب کاهش املاح زایی و بهبود کارآیی آلفا گلوکوزیداز اسیدآمیز انسان در مدل موشهای پومپ
موضوعات مرتبط
علوم پزشکی و سلامت داروسازی، سم شناسی و علوم دارویی اکتشاف دارویی
چکیده انگلیسی
Enzyme replacement therapy with recombinant human acid α-glucosidase (rhGAA) is complicated by the formation of anti-rhGAA antibodies, a short circulating half-life, instability in the plasma, and limited uptake into target tissue. Previously, we have demonstrated that phosphatidylinositol (PI) containing liposomes can reduce the immunogenicity and extend plasma survival of factor VIII (FVIII) in a mouse model of hemophilia A. In this article, we investigate the ability of PI liposomes to be used as a delivery vehicle to overcome the issues that complicate therapy with rhGAA. In a murine model of Pompe disease, administration of PI-rhGAA mitigated the immunogenicity of rhGAA, resulting in a significantly lower formation of anti-rhGAA antibodies. PI-rhGAA also showed minimal improvements to the pharmacokinetic parameters and efficacy measures compared to free rhGAA. Overall, these data suggest that PI-rhGAA may have the potential to be a useful therapeutic option for improving the treatment of Pompe disease.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Journal of Pharmaceutical Sciences - Volume 107, Issue 3, March 2018, Pages 831-837
نویسندگان
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