Leptin Therapy in People with Congenital Leptin Deficiency

摘要: Leptin is an adipokine synthesized mainly by the white adipose tissue, with a key role in regulation of food intake and energy balance. In humans, leptin deficiency observed cases lipodystrophy, functional hypothalamic amenorrhea, congenital (CLD) due to mutations gene. To date, 34 CLD have been described literature, eight different gene identified so far. For these patients, replacement therapy metreleptin (a recombinant analog) only available therapy, which has offered most patients. The analysis effects patients shown that diverse effects, not leading substantial weight loss decreases intake, but also endocrine, metabolic, immune, both structural changes brain. A better understanding physiological roles may lead development leptin-based therapies for more prevalent disorders such as fatty liver disease neurocognitive diseases.