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Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

Recombinase-activating gene-1 (RAG1)-deficient severe combined immunodeficiency (SCID) patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. Gene therapy is an alternative for those RAG1-SCID patients who lack a suitable bone marrow donor. We d...

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Detalles Bibliográficos
Autores principales:	Garcia-Perez, Laura, van Eggermond, Marja, van Roon, Lieke, Vloemans, Sandra A., Cordes, Martijn, Schambach, Axel, Rothe, Michael, Berghuis, Dagmar, Lagresle-Peyrou, Chantal, Cavazzana, Marina, Zhang, Fang, Thrasher, Adrian J., Salvatori, Daniela, Meij, Pauline, Villa, Anna, Van Dongen, Jacques J.M., Zwaginga, Jaap-Jan, van der Burg, Mirjam, Gaspar, H. Bobby, Lankester, Arjan, Staal, Frank J.T., Pike-Overzet, Karin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7163047/ https://www.ncbi.nlm.nih.gov/pubmed/32322605 http://dx.doi.org/10.1016/j.omtm.2020.03.016

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