Protein misfolding and human disease

N Gregersen, P Bross, S Vang… - Annu. Rev. Genomics …, 2006 - annualreviews.org
Protein misfolding is a common event in living cells. In young and healthy cells, the
misfolded protein load is disposed of by protein quality control (PQC) systems. In aging cells …

Disorders of mitochondrial fatty acyl‐CoA β‐oxidation

RJA Wanders, P Vreken, MEJ Den Boer… - Journal of inherited …, 1999 - Wiley Online Library
In recent years tremendous progress has been made with respect to the enzymology of the
mitochondrial fatty acid β‐oxidation machinery and defects therein. Firstly, a number of new …

Whole-genome sequencing identifies common-to-rare variants associated with human blood metabolites

T Long, M Hicks, HC Yu, WH Biggs, EF Kirkness… - Nature …, 2017 - nature.com
Genetic factors modifying the blood metabolome have been investigated through genome-
wide association studies (GWAS) of common genetic variants and through exome …

A genome-wide perspective of genetic variation in human metabolism

T Illig, C Gieger, G Zhai, W Römisch-Margl… - Nature …, 2010 - nature.com
Serum metabolite concentrations provide a direct readout of biological processes in the
human body, and they are associated with disorders such as cardiovascular and metabolic …

Tandem mass spectrometric analysis for amino, organic, and fatty acid disorders in newborn dried blood spots: a two-year summary from the New England Newborn …

TH Zytkovicz, EF Fitzgerald, D Marsden… - Clinical …, 2001 - academic.oup.com
Background: Tandem mass spectrometry (MS/MS) is rapidly being adopted by newborn
screening programs to screen dried blood spots for> 20 markers of disease in a single …

Loss of ETHE1, a mitochondrial dioxygenase, causes fatal sulfide toxicity in ethylmalonic encephalopathy

V Tiranti, C Viscomi, T Hildebrandt, I Di Meo, R Mineri… - Nature medicine, 2009 - nature.com
Ethylmalonic encephalopathy is an autosomal recessive, invariably fatal disorder
characterized by early-onset encephalopathy, microangiopathy, chronic diarrhea, defective …

Clear correlation of genotype with disease phenotype in very–long-chain Acyl-CoA dehydrogenase deficiency

BS Andresen, S Olpin, BJHM Poorthuis… - The American Journal of …, 1999 - cell.com
Very–long-chain acyl-CoA dehydrogenase (VLCAD) catalyzes the initial rate-limiting step in
mitochondrial fatty acid β-oxidation. VLCAD deficiency is clinically heterogenous, with three …

Genetic variation in an individual human exome

PC Ng, S Levy, J Huang, TB Stockwell, BP Walenz… - PLoS …, 2008 - journals.plos.org
There is much interest in characterizing the variation in a human individual, because this
may elucidate what contributes significantly to a person's phenotype, thereby enabling …

Carnitine homeostasis, mitochondrial function and cardiovascular disease

S Sharma, SM Black - Drug Discovery Today: Disease Mechanisms, 2009 - Elsevier
Carnitines are involved in mitochondrial transport of fatty acids and are of critical importance
for maintaining normal mitochondrial function. This review summarizes recent experimental …

Protein misfolding and degradation in genetic diseases

P Bross, TJ Corydon, BS Andresen… - Human …, 1999 - Wiley Online Library
Investigations of genetic diseases such as cystic fibrosis, α‐1‐antitrypsin deficiency,
phenylketonuria, mitochondrial acyl‐CoA dehydrogenase deficiencies, and many others …