The molecular basis and treatment of primary immunodeficiency disorders.
The molecular basis and treatment of primary immunodeficiency disorders.
复制标题
原发性免疫缺陷疾病的分子基础和治疗。
DOI：
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发表时间：
1997
影响因子：
3.6
通讯作者：
H. Ochs
中科院分区：
文献类型：
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作者：
B. Smart;H. Ochs
在过去的十年中，在原发性免疫缺陷疾病的分子表征和治疗方面取得了许多重要进展。这些进展包括鉴定异常基因，这些异常基因导致X连锁严重联合免疫缺陷综合征、几种形式的常染色体严重联合免疫缺陷综合征、X连锁和常染色体无丙种球蛋白血症、Wiskott-Aldrich综合征和其他原发性免疫缺陷疾病。在过去的一年中，负责这些综合征的异常基因产物的生物学功能已得到更好的定义，并导致原发性免疫缺陷疾病的新的分子缺陷也有报道。这种对原发性免疫缺陷疾病的分子基础的更好理解导致了对已建立的疗法的改进，例如基因产物替代和干细胞移植，以及新的治疗策略，例如基因疗法。
Over the past decade, a number of important advances have been made in the molecular characterization and the treatment of the primary immunodeficiency disorders. These advances include identification of the abnormal genes responsible for such syndromes as X-linked severe combined immune deficiency, several forms of autosomal severe combined immune deficiency, X-linked and autosomal agammaglobulinemia, Wiskott-Aldrich syndrome, and other primary immunodeficiency disorders. In the past year, the biologic functions of the abnormal gene products responsible for these syndromes have been better defined, and new molecular defects that lead to primary immunodeficiency disorders have also been reported. This better understanding of the molecular basis of the primary immunodeficiency disorders has led to improvement of established therapies, such as gene product replacement and stem cell transplants, and to new treatment strategies, such as gene therapy.