Pulse Brain · Growing Health Evidence Index
Tier 3 — Observational / field trialPeer-reviewed

Aberrant expression of agouti signaling protein (ASIP) as a cause of monogenic severe childhood obesity

Elena Kempf, Kathrin Landgraf, Robert Stein, Martha Hanschkow, Anja Hilbert, Rami Abou Jamra, Paula Boczki, Gunda Herberth, Andreas Kühnapfel, Yu‐Hua Tseng, Claudia Stäubert, Torsten Schöneberg, Peter Kühnen, Nigel W. Rayner, Eleftheria Zeggini, Wieland Kieß, Matthias Blüher, Antje Körner

Nature Metabolism · 2022

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Summary

This paper reports a monogenic cause of severe childhood obesity: a heterozygous tandem duplication at the ASIP locus that places the agouti signaling protein gene under control of an ubiquitously active promoter, resulting in ectopic ASIP expression across all tissues and germ layers. The phenotype observed in patients—early-onset obesity, overgrowth, red hair and hyperinsulaemia—parallels that of mutant mice expressing the homologous nonagouti gene. By rescreening a large childhood obesity cohort, the authors identified four additional patients with the identical mutation, indicating this aberrant ASIP expression is a recurrent monogenic cause of human obesity.

Regional applicability

This study was conducted in Germany using a German-based childhood obesity cohort. Whilst the genetic findings are applicable internationally given that human genetic variation is largely shared across European populations, translation to United Kingdom clinical practice would require validation in British paediatric obesity populations and assessment of prevalence in UK genetic screening cohorts.

Key measures

ASIP gene expression patterns in patient-derived pluripotent stem cells and hypothalamic-like neurons; phenotypic features (body weight, height, hair colour, insulin levels); prevalence of identical mutation in Leipzig Childhood Obesity cohort (n=1,745)

Outcomes reported

The study identified a heterozygous tandem duplication at the ASIP gene locus causing ubiquitous ectopic ASIP expression in patients with severe early-onset childhood obesity. Five patients total were identified with the identical mutation, ectopic ASIP expression, and a phenotype including obesity, overgrowth, red hair, and hyperinsulaemia.

Theme
Nutrition & health
Subject
Maternal, infant & child nutrition
Study type
Research
Study design
Case series with cohort rescreening
Source type
Peer-reviewed study
Status
Published
Geography
Germany
System type
Human clinical
DOI
10.1038/s42255-022-00703-9
Catalogue ID
SNmp6e6wxx-13mgtd

Topic tags

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