Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy.

By S. Hacein-Bey-Abina, F. Le Deist, F. Carlier, C. Bouneaud, C. Hue, J. de Villartay, A. Thrasher, N. Wulffraat, R. Sorensen, S. Dupuis-Girod and 5 more. New England Journal of Medicine, 2002

1,180 citations recorded by the source corpus.

Subfields: Genetics, Immunology

View Paper: https://www.semanticscholar.org/paper/4d965ca1c592a952cb28512a3ffff07ad000a2dd

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Genetics

1. K. High
2. J. Keith Joung
3. James Eberwine
4. K. Ozawa
5. C. Dunbar
6. J. O’Shea
7. Tae Kyung Kim
8. Michel Sadelain

In Immunology

1. J. O’Shea
2. A. Laurence
3. K. High
4. I. McInnes
5. Michel Sadelain

Citations in the corpus

• Towards safe, non-viral therapeutic gene expression in humans (2005, methodology): “Sustained transgene expression in most clinical settings has only been achieved through the use of RETROVIRAL vectors — such as the murine leukaemia virus (MLV) — which integrate their DNA into the host genome and are therefore replicated and segregated to daughter cell”
• Gene therapy comes of age (2018, positive): “Despite relatively low HSC transduction efficiencies with g-retroviral vectors, genemodified T-lineage cells were able to expand and fill the empty T cell compartment, improving immune function despiteminimal levels of genecorrected cells in other lineages (108, 109).”
• Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. (2008, methodology): “Ex vivo retroviral vector–mediated γc gene transfer into CD34+ cells resulted in successful gene therapy for SCID-X1 (1, 2).”
• The JAK-STAT Pathway: Impact on Human Disease and Therapeutic Intervention (2015, background): “This approach was effective in addressing primary immune deficiency (2, 3), but clinical trials were complicated by leukemia due to insertional oncogenesis (3, 4).”
• Gene therapy clinical trials worldwide to 2012 – an update (2013, background): “community was bolstered by the first report of successful treatment of a genetic disease by gene therapy [5,6].”
• Delivering CRISPR: a review of the challenges and approaches (2018, background): “Perhaps the most well-known early gene therapy trial involved two studies from France (Hacein-Bey-Abina et al., 2002; Hacein-Bey-Abina et al., 2010) and the UK (Gaspar et al., 2004; Gaspar et al., 2011) of children suffering from X-linked severe combined immunodeficiency (SCID X-1).”
• Mammalian cell transfection: the present and the future (2010, methodology): “For example, retrovirus murine leukemia virus (MLV) has been used as a viral vector to establish sustainable transgene expression in humans [7, 8].”