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Clinical Implications of Basic Research
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Volume 350:2302-2304 May 27, 2004 Number 22
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Targeting Gene Therapy for Osteogenesis Imperfecta
Darwin J. Prockop, M.D., Ph.D.

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Two shadows have recently been cast over the exciting field of gene therapy. One was cast by the unfortunate death of an 18-year-old man at the University of Pennsylvania in 1999.1 The patient was part of a trial in which an adenovirus containing a gene for ornithine transcarbamylase was infused into patients with partial deficiencies of the enzyme. The death highlighted the potential dangers of the direct administration of adenoviruses and, perhaps, other viruses to patients. The second shadow was cast by a clinical trial involving children with X-linked severe combined immunodeficiency (SCID).2 The trial had an added measure of . . . [Full Text of this Article]


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From the Center for Gene Therapy, Tulane University Health Sciences Center, New Orleans.


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