Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease.

By M. Cavazzana‐Calvo, S. Hacein‐Bey, G. Basile, F. Gross, E. Yvon, P. Nusbaum, F. Selz, C. Hue, S. Certain, J. Casanova and 3 more. Science, 2000

2,672 citations recorded by the source corpus.

Subfields: Genetics, Immunology

View Paper: https://www.semanticscholar.org/paper/02554c46f9829a3001a22dfe26145118b967bc15

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Citations in the corpus

Implications of the Human Genome Project for medical science. (2001, background): “There already have been promising reports of the application of gene therapy for hemophilia B(23) and severe combined immunodeficiency.(24)”
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).”
Gene therapy clinical trials worldwide to 2007—an update (2007, background): “In 2000 the morale of the community was lifted with the first report from France of successful treatment of children suffering from a rare form of X-linked severe combined immunodeficiency (SCID-X1) characterized by an early block in T and natural killer (NK) lymphocyte differentiation [5].”
Analysis of Genes and Genomes (2004, methodology): “Stem cells were collected from the bone marrow of an affected infant and treated with a retrovirus carrying a wild-type copy of the γ c gene (Cavazzana-Calvo et al., 2000).”
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].”
CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future (2020, background): “Shortly after, another gene therapy trial led to the development of leukemia in several young children induced by insertional oncogenesis from the therapy (10).”
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”
Highly efficient endogenous human gene correction using designed zinc-finger nucleases (2005, background): “Targeted alteration of DNA sequence at a human disease locus Our ultimate goal is gene correction therapy for human disease, so we set out to determine whether ZFNs can evoke a comparable increase in HR frequency at an endogenous gene and focused our efforts on the IL2Rγ gene (the protein pr”