Gambari Roberto
University of Ferrara, Department of Life Sciences and Biotechnology, Biochemistry and Molecular Biology Section , Via Fossato di Mortara n.74, 44100 Ferrara , Italy +39 532 974443 ; +39 532 974500 ;
Expert Opin Ther Pat. 2014 Mar;24(3):267-94. doi: 10.1517/13543776.2014.863874. Epub 2014 Jan 3.
DNA/RNA-based drugs are considered of major interest in molecular diagnosis and nonviral gene therapy. In this field, peptide nucleic acids (PNAs, DNA analogs in which the sugar-phosphate backbone is replaced by N-(2-aminoethyl)glycine units or similar building blocks) have been demonstrated to be excellent candidates as diagnostic reagents and biodrugs.
Recent (2002 - 2013) patents based on studies on development of PNA analogs, delivery systems for PNAs, applications of PNAs in molecular diagnosis, and use of PNA for innovative therapeutic protocols.
PNAs are unique reagents in molecular diagnosis and have been proven to be very active and specific for alteration of gene expression, despite the fact that solubility and uptake by target cells can be limiting factors. Accordingly, patents on PNAs have taken in great consideration delivery strategies. PNAs have been proven stable and effective in vivo, despite the fact that possible long-term toxicity should be considered. For possible clinical applications, the use of PNA molecules in combination with drugs already employed in therapy has been suggested. Considering the patents available and the results on in vivo testing on animal models, we expect in the near future relevant PNA-based clinical trials.
基于DNA/RNA的药物在分子诊断和非病毒基因治疗中备受关注。在该领域,肽核酸(PNA,一种DNA类似物,其磷酸糖骨架被N-(2-氨基乙基)甘氨酸单元或类似结构单元取代)已被证明是作为诊断试剂和生物药物的优秀候选物。
近期(2002 - 2013年)基于PNA类似物开发研究、PNA递送系统、PNA在分子诊断中的应用以及PNA用于创新治疗方案的专利。
PNA是分子诊断中的独特试剂,尽管溶解性和被靶细胞摄取可能是限制因素,但已被证明对基因表达改变非常有效且具有特异性。因此,关于PNA的专利非常重视递送策略。尽管应考虑可能的长期毒性,但PNA已被证明在体内稳定且有效。对于可能的临床应用,已有人建议将PNA分子与治疗中已使用的药物联合使用。考虑到现有专利以及动物模型体内测试结果,我们预计在不久的将来会有相关的基于PNA的临床试验。