Venkatapuram Vijaya Sree, Aggarwal Shagun, Kulkarni Aditya Deepak, Vineeth Venugopal Satidevi, Bhikaji Dalal Ashwin, Bhat Venkatraman, Kiran Lavanya, Patil Siddaramappa Jagdish
Department of Medical Genetics, Nizam's Institute of Medical Sciences, Hyderabad, India.
Division of Diagnostics, Centre for DNA Fingerprinting & Diagnostics, Hyderabad, India.
Am J Med Genet A. 2022 Apr;188(4):1287-1292. doi: 10.1002/ajmg.a.62622. Epub 2022 Jan 6.
Biallelic IMPAD1 pathogenic variants leads to deficiency of GPAPP (Golgi 3-prime phosphoadenosine 5-prime phosphate 3-prime phosphatase) protein and clinically causes chondrodysplasia, which is characterized by short stature with short limbs, craniofacial malformations, cleft palate, hand and foot anomalies, and various radiographic skeletal manifestations. Here we describe prenatal presentation of GPAPP deficiency caused by novel biallelic pathogenic variants, 2 base pair duplication in exon 2 of IMAPD1 gene in a patient of Asian-Indian origin. Further we report on diagnostic clues of prenatal presentation of GPAPP deficiency through ultrasonography, fetal MRI, and postmortem findings. We also provide evidence of pathophysiology of underlying GPAPP deficiency in the form of disorganization and dysplastic chondrocytes and reduced sulfation of glycoproteins through histopathology of cartilage similar to that described in mice IMPAD1 homozygous mutant model.