由此,本研究报告了使用AAV血清型8载体(GNT0003)进行基因治疗的多中心、开放标签、I/II期研究的剂量递增部分的安全性和有效性数据。 近期,来自意大利贝加莫最大的公立医院教皇若望23世医院(Papa Giovanni XXIII Hospital)的Lorenzo D’Antiga博士团队领衔...
Between 1986-1994, 12 patients (8 males and 4 females) were diagnosed at our hospital with CN syndrome. Jaundice was detected in the first few days of life in all but one, in whom detection was delayed for two weeks and resulted in kernicterus. Exchange transfusions were necessary in six...
近期，来自意大利贝加莫最大的公立医院教皇若望23世医院（Papa Giovanni XXIII Hospital）的Lorenzo D’Antiga博士团队领衔在 The New England Journal of Medicine 杂志上发表文章Gene Therapy in Patients with the Crigler-Najjar Syndrome。这项小型临床研究表明，接受载体GNT0003基因治疗的患者没有报告严重不良事件。并且...
作者 张文艳 邓国宏 ZHANG Wenyan;DENG Guohong(School of Life Sciences,Chongqing University,Chongqing 404100,China;Department of Infectious Diseases,The First Affiliated Hospital of Army Medical University,Chongqing 400038,China) 机构地区 重庆大学生命科学学院 陆军军医大学第一附属医院感染科 出处 《临床肝胆...
These addi- tional exchanges were performed on the 5th hospital day. T h e total quantity of bilirubin removed during the 2 days of exchange plasmapheresis was 1,900 mg. T h e bilirubin concentration was 6 mg/100 ml at the end of the final exchange on the 5th hospital day, and was ...
In cases complicated by kernicterus or early ABE, pediatric neurologists are instrumental in managing severity and communicating prognosis details to parents. Pediatric surgeons contribute to curative treatment by liver transplantation. Hospitalized patients' outcomes are influenced by hospital protocols and th...
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Methods A retrospective analysis was performed for the clinical data of 138 patients with GS or CN-2 who attended Beijing YouAn Hospital, Capital Medical University, from January 1, 2010 to December 31, 2019, with 109 patients in the GS group and 29 patients in the CN-2 group, and the ...
Between 1986-1994, 12 patients (8 males and 4 females) were diagnosed at our hospital with CN syndrome. Jaundice was detected in the first few days of life in all but one, in whom detection was delayed for two weeks and resulted in kernicterus. Exchange transfusions were necessary in six...
Seirei Hospital, Showa-ku, Nagoya 466, Japan 6Departmentof Pediatrics, Toho UniversitySchool of Medicine and OhashiHospital, Ohashi, Meguro-ku, Tokyo 153, Japan Key Words mutation, UDP-glucuronosyltransferase, bilirubin, hered- itary disease, jaundice Crigler-Najjar syndrome type I (CN-I) is ...