کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
3797784 1244279 2014 5 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Terapia génica en el tratamiento de los errores congénitos del metabolismo
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پزشکی و دندانپزشکی (عمومی)
پیش نمایش صفحه اول مقاله
Terapia génica en el tratamiento de los errores congénitos del metabolismo
چکیده انگلیسی
Due to the enzymatic defect in inborn errors of metabolism, there is a blockage in the metabolic pathways and an accumulation of toxic metabolites. Currently available therapies include dietary restriction, empowering of alternative metabolic pathways, and the replacement of the deficient enzyme by cell transplantation, liver transplantation or administration of the purified enzyme. Gene therapy, using the transfer in the body of the correct copy of the altered gene by a vector, is emerging as a promising treatment. However, the difficulty of vectors currently used to cross the blood brain barrier, the immune response, the cellular toxicity and potential oncogenesis are some limitations that could greatly limit its potential clinical application in human beings.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Medicina Clínica - Volume 142, Issue 12, 16 June 2014, Pages 549-553
نویسندگان
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