Truncating mutations of MAGEL2 cause Prader-Willi phenotypes and autism

CP Schaaf, ML Gonzalez-Garay, F Xia, L Potocki… - Nature …, 2013 - nature.com
CP Schaaf, ML Gonzalez-Garay, F Xia, L Potocki, KW Gripp, B Zhang, BA Peters
Nature genetics, 2013nature.com
Prader-Willi syndrome (PWS) is caused by the absence of paternally expressed, maternally
silenced genes at 15q11-q13. We report four individuals with truncating mutations on the
paternal allele of MAGEL2, a gene within the PWS domain. The first subject was ascertained
by whole-genome sequencing analysis for PWS features. Three additional subjects were
identified by reviewing the results of exome sequencing of 1,248 cases in a clinical
laboratory. All four subjects had autism spectrum disorder (ASD), intellectual disability and a …
Abstract
Prader-Willi syndrome (PWS) is caused by the absence of paternally expressed, maternally silenced genes at 15q11-q13. We report four individuals with truncating mutations on the paternal allele of MAGEL2, a gene within the PWS domain. The first subject was ascertained by whole-genome sequencing analysis for PWS features. Three additional subjects were identified by reviewing the results of exome sequencing of 1,248 cases in a clinical laboratory. All four subjects had autism spectrum disorder (ASD), intellectual disability and a varying degree of clinical and behavioral features of PWS. These findings suggest that MAGEL2 is a new gene causing complex ASD and that MAGEL2 loss of function can contribute to several aspects of the PWS phenotype.
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