Rare Genetic Diseases with Human Lean and/or Starvation Phenotype Open New Avenues for Obesity and Type II Diabetes Treatment

Author(s): Itzik Harosh

Journal Name: Current Pharmaceutical Biotechnology

Volume 14 , Issue 13 , 2013

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Treatments of obesity and type II diabetes target often gene functions involved in appetite-satiety, fat and carbohydrate metabolism or thermogenesis. None of these, have provided efficient drug therapy due to a large number of genes involved in weight and energy management, the redundancy of biochemical pathways and the environmental factors. Here I discuss a new approach based on studies of genes/proteins that are associated with human “lean or starvation" phenotype that became very rare in the course of evolution. This approach has led to the identification of the congenital enteropeptidase deficiency gene and the Andersonʼs Disease gene as a potential targets for obesity and type II diabetes treatment. The advantages of these targets are: 1) they are expressed exclusively in the intestine; 2) they are peripheral targets as opposed to systemic targets; 3) they are not redundant targets. These targets open new hopes for the development of novel drugs for the treatment of common metabolic syndrome.

Keywords: Diabetes treatment, drug discovery, lean phenotype, metabolic disorders, obesity, starvation phenotype, type II diabetes.

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Article Details

Year: 2013
Page: [1093 - 1098]
Pages: 6
DOI: 10.2174/1389201015666140408124559
Price: $65

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