TY - JOUR
T1 - Novel contiguous gene deletion in peruvian girl with Trichothiodystrophy type 4 and glutaric aciduria type 3
AU - La Serna-Infantes, Jorge
AU - Pastor, Miguel Chávez
AU - Trubnykova, Milana
AU - Velásquez, Félix Chavesta
AU - Sotomayor, Flor Vásquez
AU - Barriga, Hugo Abarca
N1 - Publisher Copyright:
© 2018 Elsevier Masson SAS
PY - 2018/7
Y1 - 2018/7
N2 - 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.
AB - 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.
KW - Chromosome microarray analysis
KW - Glutaric aciduria 3
KW - Glutaryl-CoA oxidase deficiency
KW - MLKIP
KW - Nonphotosensitive
KW - SUGCT
KW - Trichothiodystrophy
UR - http://www.scopus.com/inward/record.url?scp=85042196535&partnerID=8YFLogxK
U2 - 10.1016/j.ejmg.2018.02.004
DO - 10.1016/j.ejmg.2018.02.004
M3 - Artículo
C2 - 29421601
AN - SCOPUS:85042196535
SN - 1769-7212
VL - 61
SP - 388
EP - 392
JO - European Journal of Medical Genetics
JF - European Journal of Medical Genetics
IS - 7
ER -