Soffer Omri David, Sidlow Richard
Department of Pediatrics, Staten Island University Hospital, Staten Island, New York, New York.
Department of Pediatrics and Internal Medicine, State University of New York Health Science Center at Brooklyn, Brooklyn, New York.
Am J Med Genet A. 2016 Jul;170(7):1881-3. doi: 10.1002/ajmg.a.37665. Epub 2016 Apr 19.
Specific mutations in MECP2 cause Rett syndrome (RTT) in females whereas other mutations in the same gene cause several other syndromes in males, including X-linked intellectual disability (with and without spasticity) (OMIM 300055) and X-linked intellectual disability due to increased dosage of MECP2 (OMIM 300260). Males can also manifest an entity known as MECP2-related severe neonatal encephalopathy whose mutations are identical to those in females with RTT. We describe here the first case of MECP2-related severe neonatal encephalopathy caused by a mutation in exon one of MECP2, a mutation rarely identified in females with RTT. © 2016 Wiley Periodicals, Inc.
MECP2基因的特定突变会导致女性患雷特综合征(RTT),而同一基因的其他突变则会导致男性出现其他几种综合征,包括X连锁智力障碍(伴或不伴痉挛)(OMIM 300055)以及由于MECP2剂量增加导致的X连锁智力障碍(OMIM 300260)。男性也可能表现出一种名为MECP2相关严重新生儿脑病的病症,其突变与患RTT的女性相同。我们在此描述了首例由MECP2外显子1突变引起的MECP2相关严重新生儿脑病病例,这种突变在患RTT的女性中很少见。© 2016威利期刊公司