Hepatocyte Transplantation in Special Populations: Clinical Use in Children

Khan, Zahida; Strom, Stephen C.

doi:10.1007/978-1-4939-6506-9_1

Cited by 14 publications

(5 citation statements)

References 62 publications

(37 reference statements)

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“…Beneficial results included reduction in inflammatory response, fibrosis, and apoptotic death of hepatocytes, with improved hepatic glycogen storage and serum glucose levels [ 85 ]. In theory, cell infusions are by far safer, less invasive, and more cost effective than transplanting a whole organ; however, considerable variability exists among cell sources, preparations, and clinical protocols [ 86 ]. In practice, a single case report of human hepatocyte transplantation in a cirrhotic child with A1ATD resulted in orthotopic liver transplantation 2 days later, suggesting that cell therapy may be more appropriate as a “bridge” to organ transplant rather than a cure [ 87 ].…”

Section: Evolving Therapiesmentioning

confidence: 99%

Liver Disease in Alpha-1 Antitrypsin Deficiency: Current Approaches and Future Directions

Mitchell

Khan

2017

Curr Pathobiol Rep

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Purpose of ReviewThe aim of the study is to review the liver disease caused by alpha-1 antitrypsin deficiency (A1ATD), including pathogenesis, epidemiology, diagnostic testing, and recent therapeutic developments.Recent FindingsTherapeutic approaches target several intracellular pathways to reduce the cytotoxic effects of the misfolded mutant globular protein (ATZ) on the hepatocyte. These include promoting ATZ transport out of the endoplasmic reticulum (ER), enhancing ATZ degradation, and preventing ATZ globule-aggregation.SummaryA1ATD is the leading genetic cause of liver disease among children. It is a protein-folding disorder in which toxic insoluble ATZ proteins aggregate in the ER of hepatocytes leading to inflammation, fibrosis, cirrhosis, and increased risk of hepatocellular carcinoma. The absence of the normal A1AT serum protein also predisposes patients to pan lobar emphysema as adults. At this time, the only approved therapy for A1ATD-associated liver disease is orthotopic liver transplantation, which is curative. However, there has been significant recent progress in the development of small molecule therapies with potential both to preserve the native liver and prevent hepatotoxicity.

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Section: Evolving Therapiesmentioning

confidence: 99%

Liver Disease in Alpha-1 Antitrypsin Deficiency: Current Approaches and Future Directions

Mitchell

Khan

2017

Curr Pathobiol Rep

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“…This can be accessed by puncturing the portal vein with a catheter. In younger patients the liver can also be accessed through the umbilical vein (56). When the liver architecture is altered as in the patients with cirrhosis, hepatocyte transplantation may lead to embolization of the portal veins and portal hypertension.…”

Section: Site Of Administrationmentioning

confidence: 99%

Evaluation of different routes of administration and biodistribution of human amnion epithelial cells in mice

et al. 2019

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“…The cells are injected into the recipient through portal vein, peripheral vein [30], and intraspleenic [32] or intraperitoneal route. To enhance the transplantation efficiency, conditioning of recipient liver with partial hepatectomy [33,34], liver irradiation [35,36], or portal embolization [37] has been recently proposed. Broadly, cells are categorized into two main categories; stem cells and mature hepatocytes are the potential cell-based therapies adapted to date in the cure and regeneration of liver cirrhosis [5].…”

Section: Cell-based Therapies For Regeneration Of Liver Cirrhosismentioning

confidence: 99%

Regenerative Medicine in Liver Cirrhosis: Promises and Pitfalls

Tayyeb¹,

Azam²,

Nisar³

et al. 2017

Liver Cirrhosis - Update and Current Challenges

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Liver cirrhosis is irreversible and mostly ends up with complete loss of liver function/ end-stage liver failure, and the only proven treatment is liver transplantation. Scarcity of donor, high cost, lifelong immunosuppression, and surgical complications are the major issues associated with liver transplantation and these urge to look for alternate therapeutic approaches. Advancements in the field of regenerative medicine are arising hope for the treatment of liver cirrhosis. This chapter deals with the scope of liver regenerative medicine in the treatment of liver cirrhosis. Review of the literature showed that liver regenerative medicine no doubt holds great promises and added a lot of hope to the cure of liver diseases. Primarily, cell-based therapies had shown great potential to treat liver cirrhosis. Successful clinical human trials further strengthen their significance in the field. However, recent trends in liver regenerative medicine are focusing on the development of tissue engineering leading to generation of the whole organ. Despite advantages, liver regenerative medicine has several limitations and sometimes been over-optimistically interpreted. In conclusion, the current scenario advocates to conduct more preclinical and clinical trials to effectively replace liver transplantation with liver regenerative medicine to treat liver diseases.

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Hepatocyte Transplantation in Special Populations: Clinical Use in Children

Cited by 14 publications

References 62 publications

Liver Disease in Alpha-1 Antitrypsin Deficiency: Current Approaches and Future Directions

Liver Disease in Alpha-1 Antitrypsin Deficiency: Current Approaches and Future Directions

Evaluation of different routes of administration and biodistribution of human amnion epithelial cells in mice

Regenerative Medicine in Liver Cirrhosis: Promises and Pitfalls

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