A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency.

By S. Hacein-Bey-Abina, C. von Kalle, M. Schmidt, F. Le Deist, N. Wulffraat, Elisabeth McIntyre, I. Radford, J. Villeval, C. Fraser, M. Cavazzana‐Calvo and 1 more. New England Journal of Medicine, 2003

1,926 citations recorded by the source corpus.

Subfields: Genetics, Immunology, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/cba7d43412c1709861aea218e4cba2240921668e

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In Genetics

1. M. Kay
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Citations in the corpus

• The JAK-STAT Pathway: Impact on Human Disease and Therapeutic Intervention (2015, background): “(4) The STAT-STAT dimer translocates to the nucleus, where it can directly bind DNA and regulate gene expression.”
• Carbon Nanotubes in Biology and Medicine: In vitro and in vivo Detection, Imaging and Drug Delivery (2009, background): “ delivery method has shown promise in animal models as well as clinical trials, the safety concern of viral vectors is signficant. It is thus important to develop non-viral vectors for siRNA delivery [137, 138]. With a cleavable disulfide bond linkage between siRNA and SWNTs, we successfully deliver”
• Gene therapy clinical trials worldwide to 2007—an update (2007, background): “Following the serious adverse events in 1999 and 2002 [3,6] the momentum slowed as several”
• Progress and problems with the use of viral vectors for gene therapy (2003, background): “Recent evidence from a number of separate studies has challenged our perceptions about the risks of using integrating retrovirus vectors for certain types of gene therap”
• Chimeric Receptor mRNA Transfection as a Tool to Generate Antineoplastic Lymphocytes (2008, neutral): “However, these methods carry the risk of irreversible genomic alterations resulting from exogenous DNA integration (Hacein-Bey-Abina et al. , 2003).”
• Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “Overshadowing this impressive success, 30 months out from treatment, one of the initial patients in the French trial developed a T cell leukaemia, which was the direct result of the gene transfer vector used [10].”
• Towards safe, non-viral therapeutic gene expression in humans (2005, background): “highlighted the high risk of insertional mutagenesis: 3 of 11 treated children developed a type of T-cell leukaemia (Ref”