Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer.

By S. Raper, N. Chirmule, F. Lee, N. Wivel, A. Bagg, G. Gao, James M. Wilson, M. Batshaw. Molecular Genetics and Metabolism, 2003

1,470 citations recorded by the source corpus.

Subfields: Biotechnology, Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/7c331a19e74e6ca5a3a70bfbac1f94c38937d45e

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In Molecular Biology

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Citations in the corpus

CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future (2020, background): “The adenovirus vector triggered a much stronger immune response in Jesse than it had in other patients, causing a chain of multiple organ failures that ultimately led to his death (8).”
Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA (2011, neutral): “Current gene therapy methods in clinical trials use viral vectors that can activate innate immune sensors (32) and integrate into host cell DNA (33) with potentially serious complications.”
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges (2011, neutral): “First, AAV vectors had never been administered to liver in human subjects before, and the only death in a gene therapy trial at that point had been in a trial in which adenoviral vectors were administered to the liver in subjects with OTC deficienc”
Normothermic ex vivo lung perfusion in clinical lung transplantation. (2011, background): “Finally, we demonstrated that the isolation of donor lungs from the circulating immune system during EVLP mitigated vector-associated inflammation, a major concern of gene therapy [36, 37].”
Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “Following the serious adverse events in 1999 and 2002 [ 3 ,6] the momentum slowed as several regulatory agencies put a temporary hold on new or ongoing trials.”
Adenovirus serotype 5 hexon mediates liver gene transfer. (2008, background): “…Centre and Haemostasis Unit, Royal Free and University College Medical School, London NW3 2PF, UK 2Haemostasis and Thrombosis, MRC Clinical Sciences Centre, Imperial College London, Du Cane Road, London W12 0NN, UK 3Medical Research Council Virology Unit, University of Glasgow, Church…”
Gene therapy clinical trials worldwide to 2007—an update (2007, background): “The first major adverse event of a gene therapy treatment, the death of 18-year-old Jesse Gelsinger, who suffered from ornithine transcarbamylase (OTC) deficiency, occurred on September 17, 1999 [3].”
Immune responses to AAV vectors: overcoming barriers to successful gene therapy. (2013, neutral): “27,28 AAV vectors, conversely, have gained wide acceptance as in vivo gene transfer vectors due to their very mild proin ﬂ ammatory pro ﬁ le.”