Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

By S. Hacein-Bey-Abina, A. Garrigue, Gary P. Wang, J. Soulier, A. Lim, Estelle Morillon, E. Clappier, L. Caccavelli, É. Delabesse, K. Beldjord and 19 more. Journal of Clinical Investigation, 2008

1,797 citations recorded by the source corpus.

Subfields: Plant Science

View Paper: https://www.semanticscholar.org/paper/4b95f1e34e06001fac9cfce7ce46b89df02115ef

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Citations in the corpus

• Practice parameter for the diagnosis and management of primary immunodeficiency. (2015, background): “Gene therapy of PIDDs has met with some success, but the unexpected complication of T-cell malignancy in patients with X-linked severe combined immunodeficiency (XSCID) has tempered the initial successful results.(33,67) However, gene therapy for adenosine deaminase (ADA)–SCID has also been very suc”
• Molecular mechanisms and potential therapeutical targets in Huntington's disease. (2010, background): “Another major problem is the risk of accidental insertional mutagenesis by viral vectors and subsequent tumor formation (234).”
• Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T-cells (2012, methodology): “The durable persistence of the CD4ζ T-cells provided a unique opportunity to interrogate selection for preferred gammaretroviral integration sites over time in T cells, resulting from modulation of cellular growth control genes as a result of integration-mediated events such as distal effects from t”
• Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy (2013, background): “(HSPCs) has the potential to trigger leukemia through insertionalmutagenesis (14, 18).”
• Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease (2010, background): “Establishment of a novel human myeloid leukaemia cell line (HNT-34) with t(3;3)(q21;q26), t(9;22)(q34;q11) and the expression of EVI1 gene, P210 and P190 BCR/ABL chimaeric transcripts from a patient with AML after MDS with 3q21q26 syndrome.”
• Toxicities of chimeric antigen receptor T cells: recognition and management. (2016, neutral): “Hypothetically, the gene-therapy vector could be capable of autonomous viral replication or cause a secondary malignancy through insertional mutagenesis.(41) Importantly, neither of these toxicities involving the gene-therapy vector have been reported in clinical trials of genetically-modified T cel”
• Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “A further three patients in the French trial [3] and one in the British trial for SCID-X1 [4] went on to develop Tcell leukaemia, with four of the five patients being successfully treated with chemotherapy and in complete remission.”