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基因疗法:取得了一些成果,但仍有许多问题有待解决。

Gene therapy: some results, many problems to solve.

作者信息

Fischer A

机构信息

INSERM Unit 429, Hĵpital Necker-Enfants Malades, Paris, France.

出版信息

Cell Mol Biol (Noisy-le-grand). 2001 Dec;47(8):1269-75.

Abstract

Gene therapy is raising incredible hopes. The prospects of treating numbers of severe pathologies (hereditary, cancerous, degenerative or infectious) are vast. Nevertheless, the technological bolts to lift are still numerous, whether they be bringing the vectors into focus, the systems of expression of transgenes or the neutralization of immune responses of the host against the vector, the product of transgenes, or the knowledge of the considered pathologies of physiopathology. Solving these difficulties entails the gathering of multiple disciplines, from chemistry to medicine, passing through virology and immunology.

摘要

基因治疗正带来极大的希望。治疗多种严重病症(遗传性、癌症、退行性或传染性)的前景广阔。然而,需要攻克的技术难题仍然众多,无论是聚焦载体、转基因表达系统,还是中和宿主针对载体、转基因产物的免疫反应,亦或是对所研究病症的病理生理学的了解。解决这些难题需要汇聚从化学到医学等多个学科,还要经过病毒学和免疫学。

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