Gene Therapy for Pediatric Neurologic Disease.

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第一作者： Lauren,Jimenez-Kurlander

第一单位： Department of Pediatric Hematology and Oncology, Boston Children's Hospital, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA.

作者： Lauren,Jimenez-Kurlander ^[1] ; Christine N,Duncan ^[2]

作者单位： Department of Pediatric Hematology and Oncology, Boston Children's Hospital, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA. ^[1] Department of Pediatric Hematology and Oncology, Boston Children's Hospital, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, USA. Electronic address: Christine_Duncan@dfci.harvard.edu. ^[2]

关键词 Ex?vivo Gene therapy In?vivo Leukodystrophies Lysosomal storage disorders Motor neuron disease Neurologic disorders Peroxisomal storage disorders

主题词儿童(Child);造血干细胞移植(Hematopoietic Stem Cell Transplantation);造血干细胞(Hematopoietic Stem Cells);人类(Humans);溶酶体贮积病(Lysosomal Storage Diseases);神经系统疾病(Nervous System Diseases)

DOI 10.1016/j.hoc.2022.05.003

PMID 35760664

发布时间 2022-09-21

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