Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?

This publication has 71 references indexed in Scilit:

• Major role of local immune responses in antibody formation to factor IX in AAV gene transfer
  Gene Therapy, 2005
• Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences
  PLoS Biology, 2004
• Progress and problems with the use of viral vectors for gene therapy
  Nature Reviews Genetics, 2003
• A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency
  New England Journal of Medicine, 2003
• Site-specific genomic integration produces therapeutic Factor IX levels in mice
  Nature Biotechnology, 2002
• Dangerous liaisons: the role of “danger” signals in the immune response to gene therapy
  Blood, 2002
• Influence of Vector Dose on Factor IX-Specific T and B Cell Responses in Muscle-Directed Gene Therapy
  Human Gene Therapy, 2002
• Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy.
  Journal of Clinical Investigation, 1998
• Thirty-five years of isolated limb perfusion for melanoma: Indications and results
  British Journal of Surgery, 1996
• Cause of the ‘inhibitor’ phenotype in the haemophilias
  Nature, 1986