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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2023
Materias:
Biomedicine and Life Sciences
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

Internet

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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