کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
3466094 1596551 2014 5 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Cystic fibrosis — What are the prospects for a cure?
ترجمه فارسی عنوان
فیبروز کیستیک چشم انداز درمان چیست؟
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پزشکی و دندانپزشکی (عمومی)
چکیده انگلیسی


• A cure for cystic fibrosis is now a realistic prospect.
• Ion channel modulators and gene therapy lead the way.
• A landmark gene therapy trial using a novel vector is in progress.

Significant improvements in the treatment of cystic fibrosis over the last few decades have altered this lethal disease in children to a multisystem disorder with survival into adult life now common. In most developed countries the numbers of adult cystic fibrosis patients outnumber children. This is mainly due to improvements in care during early life. The principal cause of morbidity and mortality is pulmonary disease, and so the focus of new treatments has targeted the lungs. Identification of the underlying gene defect in the cystic fibrosis transmembrane conductance regulator has ushered in a new era in cystic fibrosis research, with prospects of a cure. In this article, we review the most exciting recent advances that correct defects in cellular processing, chloride channel function and gene therapy.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: European Journal of Internal Medicine - Volume 25, Issue 9, November 2014, Pages 803–807
نویسندگان
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