KEGG   DISEASE: 小脳萎縮を伴う小児発症神経変性疾患
エントリ  
H02738                                                             
名称    
小脳萎縮を伴う小児発症神経変性疾患
概要    
Childhood-onset neurodegeneration with cerebellar atrophy (CONDCA) is a lower motor neuron disorder due to biallelic loss-of-function mutations in AGTPBP1. Patients generally presented with muscular hypotonia, global developmental delays, and cerebellar atrophy on brain imaging from early infancy, followed by progressive worsening of neurological function. AGTPBP1 encodes cytosolic carboxypeptidase 1 (CCP1) involved in tubulin deglutamylation.
カテゴリ  
神経系疾患
階層分類  
ICD-11 による疾患分類 [BR:jp08403]
 08 神経系の疾患
  神経系のその他の疾患
   8E4Y  その他の明示された神経系の疾患
    H02738  小脳萎縮を伴う小児発症神経変性疾患
病因遺伝子 
(CONDCA) AGTPBP1 [HSA:23287] [KO:K23435]
リンク   
ICD-11: 8E4Y
OMIM: 618276
文献    
  著者
Shashi V, Magiera MM, Klein D, Zaki M, Schoch K, Rudnik-Schoneborn S, Norman A, Lopes Abath Neto O, Dusl M, Yuan X, Bartesaghi L, De Marco P, Alfares AA, Marom R, Arold ST, Guzman-Vega FJ, Pena LD, Smith EC, Steinlin M, Babiker MO, Mohassel P, Foley AR, Donkervoort S, Kaur R, Ghosh PS, Stanley V, Musaev D, Nava C, Mignot C, Keren B, Scala M, Tassano E, Picco P, Doneda P, Fiorillo C, Issa MY, Alassiri A, Alahmad A, Gerard A, Liu P, Yang Y, Ertl-Wagner B, Kranz PG, Wentzensen IM, Stucka R, Stong N, Allen AS, Goldstein DB, Schoser B, Rosler KM, Alfadhel M, Capra V, Chrast R, Strom TM, Kamsteeg EJ, Bonnemann CG, Gleeson JG, Martini R, Janke C, Senderek J
  タイトル
Loss of tubulin deglutamylase CCP1 causes infantile-onset neurodegeneration.
  雑誌
EMBO J 37:embj.2018100540 (2018)
DOI:10.15252/embj.2018100540
文献    
  著者
Karakaya M, Paketci C, Altmueller J, Thiele H, Hoelker I, Yis U, Wirth B
  タイトル
Biallelic variant in AGTPBP1 causes infantile lower motor neuron degeneration and cerebellar atrophy.
  雑誌
Am J Med Genet A 179:1580-1584 (2019)
DOI:10.1002/ajmg.a.61198
LinkDB    

» English version

DBGET integrated database retrieval system