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Therapeutic hepatocyte transplant for inherited metabolic disorders: functional considerations, recent outcomes and future prospects
Author(s) -
Vogel Kara R.,
Kennedy Andrew A.,
Whitehouse Luke A.,
Gibson K. Michael
Publication year - 2014
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/s10545-013-9656-5
Subject(s) - economic shortage , medicine , intensive care medicine , transplantation , disease , bridge to transplantation , organ transplantation , bioinformatics , metabolic disease , stem cell , heart transplantation , pathology , biology , linguistics , philosophy , genetics , government (linguistics)
The applications, outcomes and future strategies of hepatocyte transplantation (HTx) as a corrective intervention for inherited metabolic disease (IMD) are described. An overview of HTx in IMDs, as well as preclinical evaluations in rodent and other mammalian models, is summarized. Current treatments for IMDs are highlighted, along with short‐ and long‐term outcomes and the potential for HTx to supplement or supplant these treatments. Finally, the advantages and disadvantages of HTx are presented, highlighted by long‐term challenges with interorgan engraftment and expansion of transplanted cells, in addition to the future prospects of stem cell transplants. At present, the utility of HTx is represented by the potential to bridge patients with life‐threatening liver disease to organ transplantation, especially as an adjuvant intervention where severe organ shortages continue to pose challenges.