Safety and efficacy of gene therapy for RAG1-deficient SCID.
作者信息
Staal Frank J T, Pike-Overzet Karin, de Kivit Sander, Ott de Bruin Lisa, Mamede Lucia, Pergent Martine, Prevot Johan, Rothe Michael, Schambach Axel, Lankester Arjan
机构信息
Leiden University Medical Center, Leiden, the Netherlands.
Leiden University Medical Center, Leiden, the Netherlands.
出版信息
Mol Ther. 2025 May 7;33(5):1869-1870. doi: 10.1016/j.ymthe.2025.02.044. Epub 2025 Mar 18.
Abstract
摘要
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