Hershfield Michael S
Box 3049, Rom 418 Sands Building, Duke University Medical Center, Durham, NC 27710, USA.
Curr Opin Immunol. 2003 Oct;15(5):571-7. doi: 10.1016/s0952-7915(03)00104-3.
Adenosine deaminase (ADA) deficiency is associated with a broad clinical and mutational spectrum. Defining the relationship of genotype to phenotype among patients with different degrees of immunodeficiency has been complicated because the disease is rare, most mutations are 'private' and patients are often heteroallelic. In recent years, knowledge of ADA structure and systematic expression of mutant alleles have revealed that phenotype is strongly associated with the sum of ADA activity provided by both alleles. A scale for ranking novel ADA alleles based on expression may have utility if newborn screening for primary immunodeficiency disorders is initiated.
腺苷脱氨酶(ADA)缺乏症与广泛的临床和突变谱相关。由于该疾病罕见,大多数突变是“私人的”,且患者通常为杂合等位基因,因此确定不同程度免疫缺陷患者的基因型与表型之间的关系一直很复杂。近年来,对ADA结构的了解以及突变等位基因的系统表达表明,表型与两个等位基因提供的ADA活性总和密切相关。如果启动原发性免疫缺陷疾病的新生儿筛查,基于表达对新的ADA等位基因进行排名的量表可能会有用。