Biological therapies for inherited diseases: social and bioethical considerations. Hemophilia as an example
作者： Antonio Liras
DOI: 10.1517/14712598.2015.1029451
关键词:
摘要: Introduction: In hemophilia, advanced therapies are warranted from a conceptual and methodological standpoint. Current therapy strategies centered on the use of adeno-associated viral vectors, although problems related to immunogenicity hepatotoxicity still remain.Areas covered: Future clinical trials will have scrupulously observe international bioethical standards in terms patient selection, particularly children. Patient recruitment rates likely remain low due stringent exclusion criteria usually imposed trial population regarding their hepatic immunological markers presence coinfection; existence an optimal palliative treatment.Expert opinion: Accordingly, results obtained be statistical significance, which could hinder application practice. Another important issue is degree society embraces these new emerging therapies. The unfamiliarity with meth...
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