Mucolipidosis type II, also known as I-cell disease, is an autosomal recessive inborn error of metabolism, resulting from loss-of-function mutations in GNPTAB. Affected infants exhibit multiple physical anomalies and developmental delay, and death from disease follows in early childhood. Here we present an instructive case of mucolipidosis type II affecting 1 fetus and placental disk in a dichorionic-diamnionic twin pregnancy delivered at 36-wk gestation. The second twin and placental disk showed no abnormality. On microscopic examination, the affected placenta displayed marked vacuolization of the syncytiotrophoblast and Hofbauer cells, which was confirmed on ultrastructural examination. To our knowledge, this is the first description of placental findings in a twin pregnancy, wherein only 1 twin is affected by an inborn error of metabolism. This provides an opportunity to highlight the placental abnormalities seen in this group of diseases, and to emphasize the role of pathologic examination in early detection of otherwise unsuspected inborn errors of metabolism.
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http://dx.doi.org/10.1097/PGP.0000000000000506 | DOI Listing |
BMC Pediatr
December 2024
Department of Genetics and Endocrinology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, 9 Jinsui Road, Guangzhou, 510623, China.
Background: Mucolipidosis (ML) II and III alpha/beta are lysosomal disorders caused by mutations in the GNPTAB gene which encodes the alpha and beta subunits of the heterohexameric enzyme, N-acetylglucosamine-1-phosphotransferase.
Method: To explore the clinical and molecular characteristics of the 20 ML II and III alpha/beta patients, clinical data was collected and GNPTAB gene was analyzed by nest PCR and direct Sanger-sequencing. The activity of several lysosomal enzymes was measured in the plasma.
J Biomol Struct Dyn
December 2024
Department of Biotechnology, Sri Ramachandra Institute of Higher Education and Research (DU), Chennai, India.
Mucolipidosis type IV (MLIV) is classified as an exceptionally autosomal recessive condition due to a change in MCOLN1 that encodes the mucolipin-1 protein. ML-1 is a membrane protein comprising 6 Trans regions, which are situated at the LELs, a serine lipase area, and a nuclear localization sign. The characteristic features of the ML4 patients are mental retardation and skeletal deformities due to an increase in lipid molecules in the brain, other tissues, and organs.
View Article and Find Full Text PDFJ Am Soc Nephrol
December 2024
Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Orphanet J Rare Dis
November 2024
Department of Endocrinology, Beijing Children's Hospital, Capital Medical University, National Centre for Children's Health, Genetics, Metabolism, Beijing, 100045, China.
Retin Cases Brief Rep
October 2024
Division of Ophthalmology, University of São Paulo Medical School (USP), Av. Dr Enéas de Carvalho Aguiar, 255, Cerqueira César, São Paulo, SP, 05403-001, Brazil.
Purpose: Describe a case of symmetric bilateral macular atrophy as an ophthalmological manifestation of Mucolipidosis type 3.
Methods: Multimodal retinal imaging evaluation was performed, with color fundus photograph, fundus autofluorescence, fluorescein angiography and optical coherence tomography. Genetic testing confirmed the systemic diagnosis.
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