Genetic Metabolic Unit, Department of Pediatrics, Advanced Pediatrics Centre, Post Graduate Institute of Medical Education and Research, Chandigarh, India.
Department of Hematology, Post Graduate Institute of Medical Education and Research, Chandigarh, India.
Lab Med. 2021 Sep 1;52(5):499-502. doi: 10.1093/labmed/lmaa116.
The clinical manifestations of FGFR3 sequence variations can vary from mild unnoticed short stature to neonatal lethal dwarfism and can be causative of phenotypes including achondroplasia, hypochondroplasia, and thanatophoric dysplasia. Clinical data describe an 11 month old girl with restricted growth and preserved intellect. She had rhizomelic short stature with peculiar facies but no Acanthosis nigricans. In view of the absence of the hotspot mutation c.1138 G>A/G>C (p.Gly380Arg), complete gene sequencing was done that revealed a rare sequence variation, NM_000142.4:c.1043C>G (p.Ser348Cys) in FGFR3. This sequence variation has not been reported from India so far. This report emphasizes the benefit of sequencing the whole gene in individuals who are negative for hotspot mutation of achondroplasia with strong clinical suspicion.
FGFR3 序列变异的临床表现从轻微的不易察觉的身材矮小到新生儿致死性 dwarfism 不等,可导致包括软骨发育不全、软骨发育不良和致死性发育不良等表型。临床数据描述了一名 11 个月大的女孩,其生长受限但智力正常。她患有肢端肥大性身材矮小,具有特殊的面容,但没有黑棘皮病。鉴于没有热点突变 c.1138 G>A/G>C(p.Gly380Arg),我们进行了完整的基因测序,发现了 FGFR3 中的一个罕见序列变异 NM_000142.4:c.1043C>G(p.Ser348Cys)。到目前为止,这种序列变异还没有在印度报道过。本报告强调了在具有强烈临床怀疑的软骨发育不全热点突变阴性个体中对整个基因进行测序的益处。