Perlman Syndrome: Overgrowth, Wilms Tumor Predisposition and DIS3L2

MR Morris, D Astuti, ER Maher - American Journal of Medical …, 2013 - Wiley Online Library
American Journal of Medical Genetics Part C: Seminars in Medical …, 2013Wiley Online Library
Perlman syndrome is a rare autosomal recessively inherited congenital overgrowth
syndrome characterized by polyhydramnios, macrosomia, characteristic facial
dysmorphology, renal dysplasia and nephroblastomatosis and multiple congenital
anomalies. Perlman syndrome is associated with high neonatal mortality and, survivors
have developmental delay and a high risk of Wilms tumor. Recently a Perlman syndrome
locus was mapped to chromosome 2q37 and homozygous or compound heterozygous …
Abstract
Perlman syndrome is a rare autosomal recessively inherited congenital overgrowth syndrome characterized by polyhydramnios, macrosomia, characteristic facial dysmorphology, renal dysplasia and nephroblastomatosis and multiple congenital anomalies. Perlman syndrome is associated with high neonatal mortality and, survivors have developmental delay and a high risk of Wilms tumor. Recently a Perlman syndrome locus was mapped to chromosome 2q37 and homozygous or compound heterozygous mutations were characterized in DIS3L2. The DIS3L2 gene product has ribonuclease activity and homology to the DIS3 component of the RNA exosome. It has been postulated that the clinical features of Perlman syndrome result from disordered RNA metabolism and, though the precise targets of DIS3L2 have yet to be characterized, in cellular models DIS3L2 knockdown is associated with abnormalities of cell growth and division. © 2013 Wiley Periodicals, Inc.
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