Human heterologous liver cells transiently improve hyperammonemia and ureagenesis in individuals with severe urea cycle disorders

Meyburg Jochen; Opladen Thomas; Spiekerkoetter Ute; Schlune Andrea; Schenk Jens-Peter; Schmidt Jan; Weitz Juergen; Okun Juergen; Buerger Friederike; Ben Omran Tawfeg; Abdoh Ghassan; Al Rifai Hilal; Monavari Ahmad; Konstantopoulou Vassiliki; Kolker Stefan; Yudkoff Marc; Hoffmann Georg F.

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Human heterologous liver cells transiently improve hyperammonemia and ureagenesis in individuals with severe urea cycle disorders

机译：人类异源肝细胞瞬时改善具有严重尿素循环疾病的个体中的高血肿性和尿素作用

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Background Urea cycle disorders (UCDs) still have a poor prognosis despite several therapeutic advancements. As liver transplantation can provide a cure, liver cell therapy (LCT) might be a new therapeutic option in these patients.

机译：背景技术尿素周期疾病（UCDS）仍然具有较差的预后，尽管有几种治疗进步。由于肝移植可以提供治愈，肝细胞治疗（LCT）可能是这些患者的新治疗选择。

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来源
《Journal of inherited metabolic disease 》 |2018年第1期| 共10页
作者
Meyburg Jochen; Opladen Thomas; Spiekerkoetter Ute; Schlune Andrea; Schenk Jens-Peter; Schmidt Jan; Weitz Juergen; Okun Juergen; Buerger Friederike; Ben Omran Tawfeg; Abdoh Ghassan; Al Rifai Hilal; Monavari Ahmad; Konstantopoulou Vassiliki; Kolker Stefan; Yudkoff Marc; Hoffmann Georg F.;
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作者单位

Univ Hosp Heidelberg Ctr Child &

Adolescent Med Neuenheimer Feld 430 D-69120 Heidelberg Germany;

Univ Hosp Heidelberg Ctr Child &

Adolescent Med Neuenheimer Feld 430 D-69120 Heidelberg Germany;

Heinrich Heine Univ Med Fac Dept Gen Pediat Neonatol &

Paediat Cardiol Dusseldorf Germany;

Heinrich Heine Univ Med Fac Dept Gen Pediat Neonatol &

Paediat Cardiol Dusseldorf Germany;

Univ Hosp Div Pediat Radiol Dept Diagnost &

Intervent Radiol Heidelberg Germany;

Univ Hosp Dept Visceral &

Transplant Surg Heidelberg Germany;

Univ Hosp Dept Visceral &

Transplant Surg Heidelberg Germany;

Univ Hosp Heidelberg Ctr Child &

Adolescent Med Neuenheimer Feld 430 D-69120 Heidelberg Germany;

Univ Hosp Heidelberg Ctr Child &

Adolescent Med Neuenheimer Feld 430 D-69120 Heidelberg Germany;

Hamad Med Corp Dept Pediat Doha Qatar;

Hamad Med Corp Dept Pediat Doha Qatar;

Hamad Med Corp Dept Pediat Doha Qatar;

Natl Ctr Inherited Metab Disorders Dublin Ireland;

Univ Childrens Hosp Div Pulmonol Allergol Endocrinol &

Metab Med Vienna Austria;

Univ Hosp Heidelberg Ctr Child &

Adolescent Med Neuenheimer Feld 430 D-69120 Heidelberg Germany;

Childrens Hosp Philadelphia Div Metab Dis Philadelphia PA 19104 USA;

Univ Hosp Heidelberg Ctr Child &

Adolescent Med Neuenheimer Feld 430 D-69120 Heidelberg Germany;

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正文语种 eng
中图分类内分泌腺疾病及代谢病 ;
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专利

1. Human heterologous liver cells transiently improve hyperammonemia and ureagenesis in individuals with severe urea cycle disorders [J] . Meyburg Jochen, Opladen Thomas, Spiekerkoetter Ute, Journal of inherited metabolic disease . 2018 ,第1期

机译：人类异源肝细胞瞬时改善具有严重尿素循环疾病的个体中的高血肿性和尿素作用
2. Phase I/II Trial of Liver-derived Mesenchymal Stem Cells in Pediatric Liver-based Metabolic Disorders: A Prospective, Open Label, Multicenter, Partially Randomized, Safety Study of One Cycle of Heterologous Human Adult Liver-derived Progenitor Cells (HepaStem) in Urea Cycle Disorders and Crigler-Najjar Syndrome Patients [J] . Smets Francoise, Dobbelaere Dries, McKiernan Patrick, Transplantation: Official Journal of the Transplantation Society . 2019 ,第9期

机译：肝脏衍生间充质干细胞的I / II试验在儿科肝脏代谢障碍中：尿素异源人成人肝脏祖细胞（HepaStem）的一个前瞻性，开放标记，多中心，部分随机化，安全性研究尿素循环疾病和克里克勒 - 纳吉官综合征患者
3. In-vitro modelling of urea cycle liver disorder by using human induced pluripotent stem cells [J] . Akbari S., Gurhan G., Sengun B., Human gene therapy . 2016 ,第11期

机译：利用人类诱导的多能干细胞进行尿素循环性肝病的体外建模
4. ARTIFICIAL CELLS CONTAINING GENETICALLY ENGINEERED E. COLI DH5 CELLS FOR UREA AND AMMONIA REMOVAL IN KIDNEY AND LIVER FAILURE [C] . S. Prakash, T. M. S. Chang 1995 IEEE engineering in medicine and biology 17th annual conference and 21st Canadian medical and biological engineering conference . 1997

机译：包含基因工程化大肠杆菌E.coli DH5细胞的人工细胞，用于肾脏和肝脏衰竭的尿素和氨气去除
5. Treatment Choice in Urea Cycle Disorders: How Healthcare Access Contributes to the Decision to Continue Medical Management or Pursue Liver Transplant [D] . Gerstein, Maya Tuchman. 2019

机译：尿素周期疾病治疗选择：医疗保健机会如何导致继续医疗管理或追求肝移植的决定
6. Improving Long Term Outcomes in Urea Cycle Disorders-Report from the Urea Cycle Disorders Consortium [O] . Susan Waisbren, Andrea Gropman, Mark L. Batshaw -1

机译：改善尿素循环障碍的长期结果-尿素循环障碍协会的报告
7. Phase I/II Trial of Liver–derived Mesenchymal Stem Cells in Pediatric Liver–based Metabolic Disorders: A Prospective, Open Label, Multicenter, Partially Randomized, Safety Study of One Cycle of Heterologous Human Adult Liver–derived Progenitor Cells (HepaStem) in Urea Cycle Disorders and Crigler-Najjar Syndrome Patients [O] . Françoise Smets, Dries Dobbelaere, Patrick McKiernan, 2019

机译：肝脏衍生间充质干细胞的I / II试验在儿科肝脏代谢障碍中：尿素异源人成人肝脏祖细胞（HepaStem）的一个前瞻性，开放标记，多中心，部分随机化，安全性研究尿素循环疾病和克里克勒 - 纳吉官综合征患者

Human heterologous liver cells transiently improve hyperammonemia and ureagenesis in individuals with severe urea cycle disorders

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