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Gene Therapy Death Prompts Review of Adenovirus Vector
Abstract
For the past 3 months, faculty and staff members at the University of Pennsylvania9s Institute for Human Gene Therapy have been trying to understand why a relatively fit 18-year-old with an inherited enzyme deficiency died on 17 September, 4 days after doctors at Penn injected a genetically altered virus into his liver. Last week, they presented their data to a government advisory committee. The patient was the first in a gene therapy trial to die of the therapy itself; his death is the latest blow to a field that has been struggling to live up to the promise and hype surrounding the first gene therapy trials a decade ago.
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“In another tragedy, an 18-year-old male suffering from a partial deficiency of ornithine transcarbamylase (OTC) died after developing a massive inflammatory response to the genetic cargo delivery vehicle, an adenovirus vector, four hours after receiving the treatment (Marshall, 1999; Authors, 2002).”
From Delivering CRISPR: a review of the challenges and approaches (2018) · cites this paper as Marshall, 1999
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Metadata
- Authors
- Marshall, E.
- Venue
- Science
- Published
- 1999-12-17
- Volume
- 286
- Issue
- 5448
- Pages
- 2244-2245
- ISSN
- 0036-8075
- 1095-9203
- Cited by
- 760
- References
- 0
- Language
- en
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