Mutation Study of Malaysian Patients with Ornithine Transcarbamylase Deficiency: Clinical, Molecular, and Bioinformatics Analyses of Two Novel Missense Mutations of the OTC Gene.

Ornithine transcarbamylase deficiency (OTCD), an X-linked disorder that results from mutations in the OTC gene, causes hyperammonemia and leads to various clinical manifestations. Mutations occurring close to the catalytic site of OTCase can cause severe OTCD phenotypes compared with those caused by...

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Publicado en:BioMed Research International Vol. 2018; pp. 1 - 16
Autores principales: Ali, Ernie Zuraida, Zakaria, Yuslina, Mohd Radzi, Mohd Amran, Ngu, Lock Hock, Jusoh, Siti Azma
Formato: pictorial research tables/charts Journal Article
Publicado: Wiley-Blackwell 8/5/2018
Acceso en línea:Ver este registro en EBSCOhost
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      dt: 8/5/2018
      vid: 2018
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      pub: Wiley-Blackwell
      place: Malden, Massachusetts
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        131083535
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        10.1155/2018/4320831
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        atl: Mutation Study of Malaysian Patients with Ornithine Transcarbamylase Deficiency: Clinical, Molecular, and Bioinformatics Analyses of Two Novel Missense Mutations of the OTC Gene.
      aug:
        au:
          Ali, Ernie Zuraida
          Zakaria, Yuslina
          Mohd Radzi, Mohd Amran
          Ngu, Lock Hock
          Jusoh, Siti Azma
        affil: Molecular Diagnostics and Protein Unit, Specialized Diagnostics Centre, Institute for Medical Research (IMR), Jalan Pahang, 50588 Kuala Lumpur, Malaysia
      sug:
        subj:
          Amino Acid Metabolism, Inborn Errors Familial and Genetic
          Mutation
          Bioinformatics
          Malaysia
          Human
          Male
          Female
          Polymorphism, Genetic
          Male
          Female
      ab: Ornithine transcarbamylase deficiency (OTCD), an X-linked disorder that results from mutations in the OTC gene, causes hyperammonemia and leads to various clinical manifestations. Mutations occurring close to the catalytic site of OTCase can cause severe OTCD phenotypes compared with those caused by mutations occurring on the surface of this protein. In this study, we report two novel OTC missense mutations, Q171H and N199H, found in Malaysian patients. Q171H and N199H caused neonatal onset OTCD in a male and late OTCD in a female, respectively. In silico predictions and molecular docking were performed to examine the effect of these novel mutations, and the results were compared with other 30 known OTC mutations. In silico servers predicted that Q171H and N199H, as well as 30 known missense mutations, led to the development of OTCD. Docking analysis indicated that N-(phosphonoacetyl)-L-ornithine (PALO) was bound to the catalytic site of OTCase mutant structure with minimal conformational changes. However, the mutations disrupted interatomic interactions in the catalytic site. Therefore, depending on the severity of disruption occurring at the catalytic site, the mutation may affect the efficiency of mechanism and functions of OTCase.
      pubtype: Academic Journal
      doctype:
        pictorial
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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