title: Biological therapies for inherited diseases: social and bioethical considerations. Hemophilia as an example.
abstract: BACKGROUND
  In hemophilia, advanced therapies are warranted from a conceptual and methodological standpoint. Current advanced therapy strategies are centered on the use of adeno-associated viral vectors, although problems related to immunogenicity and hepatotoxicity still remain.
  
  
  METHODS
  Future clinical trials will have to scrupulously observe international bioethical standards in terms of patient selection, particularly children. Patient recruitment rates are likely to remain low due to the stringent exclusion criteria usually imposed on the trial population regarding their hepatic and immunological markers and the presence of viral coinfection; and to the existence of an optimal palliative treatment.
  
  
  CONCLUSIONS
  Accordingly, the results obtained are likely to be of low statistical significance, which could hinder their application to clinical practice. Another important issue is the degree to which society embraces these new emerging therapies. The unfamiliarity of society with these new methods, together with the many unresolved questions about them that remain, may threaten their acceptance not only by society at large but also by health-care professionals, which would limit their translational application to clinical practice.
authors.affiliation: Complutense University of Madrid, Research Institute 12 October Hospital of Madrid, Department of Physiology , Madrid , Spain +34649907879 ; aliras@hotmail.com.
authors.forename: Antonio
authors.initials: A
authors.lastname: Liras
mesh_terms: D001691:Biological Therapy / Q000941:ethics* / Q000379:methods; D064987:Cell- and Tissue-Based Therapy / Q000941:ethics / Q000379:methods; D015316:Genetic Therapy / Q000941:ethics / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage; D006467:Hemophilia A / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D010166:Palliative Care / Q000941:ethics / Q000379:methods; D018579:Patient Selection / Q000941:ethics*; D012929:Psychological Distance
keywords: advanced therapies; cell therapy; gene therapy; hemophilia; social and bioethical