肽核酸在基因诊断和治疗中的研究进展

Progress of peptide nucleic acid in gene diagnosis and therapy

肽核酸是一种以多肽为骨架，类似核苷酸的物质。它不带电荷，能抵抗核酸酶和蛋白酶的降解；它与ＤＮＡ或ＲＮＡ杂交特异性很强，可与靶基因形成稳定的三螺旋结构。肽核酸能够抑制基因的复制、转录、逆转录和翻译过程，在基因诊断及治疗方面有着广泛的用途。

Peptide nucleic acid (PNA) is a DNA mimic with a pseudopeptide backbone. It has non-charged nature, and own highly resistant to protease and nuclease attachs; it binds to complementary RNA or DNA with specificity and form a triplex complex with highly stability. PNA not only block protein expression on the transcriptional and translational level, but also inhibit DNA replication and reverse transcription. Many applications have been explored for PNA as a new kind of molecular biological tool and a gene-targeting strategy.