Genetic modification of lymphocytes by retrovirus-based vectors.
Genetic modification of lymphocytes by retrovirus-based vectors.
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DOI：
10.1016/j.coi.2012.08.007
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发表时间：
2012-10
影响因子：
7
通讯作者：
J. D. Suerth;A. Schambach;C. Baum
中科院分区：
文献类型：
--
作者：
J. D. Suerth;A. Schambach;C. Baum
淋巴细胞的遗传修饰是新兴的基因治疗领域中的重要课题。许多针对免疫缺陷综合征或癌症的临床试验已经显示出治疗益处;进一步的应用涉及炎症和感染性疾病。逆转录病毒载体的开发需要详细了解与宿主的相互作用。大多数研究人员使用简单的γ逆转录病毒载体直接或通过造血干细胞和祖细胞修饰淋巴细胞。设计慢病毒、巨噬细胞病毒（泡沫病毒）和α逆转录病毒载体以减少细胞刺激的必要性并利用潜在更安全的整合特性。新的表面修饰（假型化）和转基因，使用合成成分构建，扩大了逆转录病毒工具箱，共同承诺增加特异性和效力。产品一致性将是常规临床使用的重要标准。
The genetic modification of lymphocytes is an important topic in the emerging field of gene therapy. Many clinical trials targeting immunodeficiency syndromes or cancer have shown therapeutic benefit; further applications address inflammatory and infectious disorders. Retroviral vector development requires a detailed understanding of the interactions with the host. Most researchers have used simple gammaretroviral vectors to modify lymphocytes, either directly or via hematopoietic stem and progenitor cells. Lentiviral, spumaviral (foamyviral) and alpharetroviral vectors were designed to reduce the necessity for cell stimulation and to utilize potentially safer integration properties. Novel surface modifications (pseudotyping) and transgenes, built using synthetic components, expand the retroviral toolbox, altogether promising increased specificity and potency. Product consistency will be an important criterion for routine clinical use.