Novel contiguous gene deletion in peruvian girl with Trichothiodystrophy type 4 and glutaric aciduria type 3
- Jorge La Serna-Infantes,
- Miguel Chávez Pastor,
- Milana Trubnykova,
- Félix Chavesta Velásquez,
- Flor Vásquez Sotomayor,
- Hospital Guillermo Almenara Irigoyen,
- Instituto Nacional de Salud del Niño,
Publication Information
Output type
Original language
EnglishPages from-to (Number of pages)
Pages 388-392 (5 pages)Journal (Volume, Issue Number)
European Journal of Medical Genetics (Volume 61, Issue 7)Publication milestones
- Published - 07/2018
Publication status
ISSN
1769-7212Publication IDs
- Scopus: 85042196535
- PubMed: 29421601
Abstract
Trichothiodystrophy type 4 is a rare autosomal recessive and ectodermal disorder, characterized by dry, brittle, sparse and sulfur-deficient hair and other features like intellectual disability, ichthyotic skin and short stature, caused by a homozygous mutation in MPLKIP gene. Glutaric aciduria type 3 is caused by a homozygous mutation in SUGCT gene with no distinctive phenotype. Both genes are localized on chromosome 7 (7p14). We report an 8-year-old female with short stature, microcephaly, development delay, intellectual disability and hair characterized for dark, short, coarse, sparse and brittle associated to classical trichorrhexis microscopy pattern. Chromosome microarray analysis showed a 125 kb homozygous pathogenic deletion, which includes genes MPLKIP and SUGCT, not described before. This is the first case described in Peru of a novel contiguous gene deletion of Trichothiodystrophy type 4 and Glutaric aciduria type 3 performed by chromosome microarray analysis, highlighting the contribution and importance of molecular technologies on diagnosis of rare genetic conditions.
