Peroxisome Assembly, Degradation, and Disease

Kim, Peter K.

doi:10.1002/9780470747919.ch13

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Htx For Inherited Metabolic Diseases1

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2013

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“…Inherited peroxisomal disorders group into either single metabolic enzyme disorders or peroxisome biogenesis disorders (PBD) (Kim 2009), with a cumulative incidence of ~1:20,000 live births. Prior to transport to the mitochondria for subsequent β-oxidation, pristanic acid is generated from phytanic acid via α-oxidation.…”

Section: Htx For Inherited Metabolic Diseasesmentioning

confidence: 99%

Therapeutic hepatocyte transplant for inherited metabolic disorders: functional considerations, recent outcomes and future prospects

Vogel¹,

Kennedy²,

Whitehouse³

et al. 2013

J of Inher Metab Disea

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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.

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Section: Htx For Inherited Metabolic Diseasesmentioning

confidence: 99%