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Integrome signatures of lentiviral gene therapy for SCID-X1 patients
Lentiviral vector (LV)–based gene therapy holds promise for a broad range of diseases. Analyzing more than 280,000 vector integration sites (VISs) in 273 samples from 10 patients with X-linked severe combined immunodeficiency (SCID-X1), we discovered shared LV integrome signatures in 9 of 10 patient...
Autores principales: | Yan, Koon-Kiu, Condori, Jose, Ma, Zhijun, Metais, Jean-Yves, Ju, Bensheng, Ding, Liang, Dhungana, Yogesh, Palmer, Lance E., Langfitt, Deanna M., Ferrara, Francesca, Throm, Robert, Shi, Hao, Risch, Isabel, Bhatara, Sheetal, Shaner, Bridget, Lockey, Timothy D., Talleur, Aimee C., Easton, John, Meagher, Michael M., Puck, Jennifer M., Cowan, Morton J., Zhou, Sheng, Mamcarz, Ewelina, Gottschalk, Stephen, Yu, Jiyang |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Association for the Advancement of Science
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10558130/ https://www.ncbi.nlm.nih.gov/pubmed/37801507 http://dx.doi.org/10.1126/sciadv.adg9959 |
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