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Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

X-linked Severe Combined Immunodeficiency (SCID-X1) due to IL2RG mutations is potentially fatal in infancy where ‘emergency’ life-saving stem cell transplant may only achieve incomplete immune reconstitution following transplant. Salvage therapy SCID-X1 patients over 2 years old (NCT01306019) is a n...

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Detalles Bibliográficos
Autores principales:	De Ravin, Suk See, Liu, Siyuan, Sweeney, Colin L., Brault, Julie, Whiting-Theobald, Narda, Ma, Michelle, Liu, Taylor, Choi, Uimook, Lee, Janet, O’Brien, Sandra Anaya, Quackenbush, Priscilla, Estwick, Tyra, Karra, Anita, Docking, Ethan, Kwatemaa, Nana, Guo, Shuang, Su, Ling, Sun, Zhonghe, Zhou, Sheng, Puck, Jennifer, Cowan, Morton J., Notarangelo, Luigi D., Kang, Elizabeth, Malech, Harry L., Wu, Xiaolin
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2022
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9240040/ https://www.ncbi.nlm.nih.gov/pubmed/35764638 http://dx.doi.org/10.1038/s41467-022-31344-x

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