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Identification of Cryptic Novel α-Galactosidase A Gene Mutations: Abnormal mRNA Splicing and Large Deletions

Anderson-Fabry (FD) disease is an inborn error of metabolism caused by a deficiency of α-galactosidase A (GLA), a lysosomal enzyme. Many male FD patients display a classic FD phenotype; however, some female patients have neither reduced leukocyte GLA enzyme activity level nor FD symptoms. Thus, GLA...

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Detaylı Bibliyografya
Yayımlandı:JIMD Rep
Asıl Yazarlar: Higuchi, Takashi, Kobayashi, Masahisa, Ogata, Jin, Kaneshiro, Eiko, Shimada, Yohta, Kobayashi, Hiroshi, Eto, Yoshikatsu, Maeda, Shiro, Ohtake, Akira, Ida, Hiroyuki, Ohashi, Toya
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Springer Berlin Heidelberg 2016
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC5110446/
https://ncbi.nlm.nih.gov/pubmed/27255140
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2015_475
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