. Author manuscript; available in PMC: 2013 Sep 1.

Published in final edited form as: Adv Drug Deliv Rev. 2012 Mar 26;64(12):1331–1340. doi: 10.1016/j.addr.2012.03.013

Table 7.

Published deaths of subjects in human gene therapy trials

Year	Disease target	Vector	Comment	Death related to gene transfer?	Ref
1999	Ornithine transcarbamylase deficiency	Adenovirus	Patient died within 4 days from cytokine storm	Yes	(18)
2002	X-linked severe combined immunodeficiency disease	Retrovirus	Leukemia developed, linked to insertion of retrovirus adjacent to oncogene	Yes	(16)
2006	X-linked chronic granulomatous disease	Retrovirus	Loss of transgene expression led to death from underlying disease	No	(148)
2007	Rheumatoid arthritis	AAV	Histoplasmosis and retroperitoneal aneurysm	No	(15)

Adapted from Evans et al., 2008(14)

AAV: Adeno-associated virus