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与体内脂质体介导的基因治疗相关的细胞毒性问题。

Cytotoxicity issues pertinent to lipoplex-mediated gene therapy in-vivo.

作者信息

Dass Crispin R

机构信息

Johnson & Johnson Research, Eveleigh, New South Wales, Australia.

出版信息

J Pharm Pharmacol. 2002 May;54(5):593-601. doi: 10.1211/0022357021778817.

DOI:10.1211/0022357021778817
PMID:12005353
Abstract

Cationic liposomes bind with nucleic acids such as plasmids and oligodeoxynucleotides to form complexes known as lipoplexes. Although these lipoplexes have several advantages over other forms of nucleic acid transfer methods in cell culture and in-vivo, toxicity remains a problem, especially in-vivo. Nevertheless, these carriers have been used in clinical trials against cystic fibrosis and cancer and their usage is attributed mainly to their versatility, especially when it comes to the range of routes available for administration of nucleic-acid-based drugs in-vivo.

摘要

阳离子脂质体与核酸(如质粒和寡脱氧核苷酸)结合形成称为脂质体复合物的复合物。尽管这些脂质体复合物在细胞培养和体内研究中相较于其他形式的核酸转移方法具有若干优势,但毒性仍然是一个问题,尤其是在体内研究中。然而,这些载体已被用于针对囊性纤维化和癌症的临床试验,其应用主要归因于它们的多功能性,特别是在体内给予基于核酸的药物时可采用的给药途径范围方面。

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