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  • Steven E. Raper, Narendra Chirmule, +5 authors Mark L. Batshaw
  • Biology, Medicine
  • Molecular genetics and metabolism
  • 2003 (First Publication: 1 September 2003)
  • We report the death of an 18-year-old male with partial ornithine transcarbamylase (OTC) deficiency who participated in a pilot (safety) study of gene therapy. The vector used for this trial wasContinue Reading
  • Alberto Auricchio, Gary P Kobinger, +5 authors Jean Bennett
  • Biology, Medicine
  • Human molecular genetics
  • 2001 (First Publication: 1 December 2001)
  • Recombinant vectors based on adeno-associated virus (AAV) or human immunodeficiency 1 (lentivirus) are promising tools for long term in vivo gene delivery. Their design allows the exchange of capsidsContinue Reading