Department of Pharmaceutics, National Institute of Pharmaceutical Education & Research (NIPER), Hyderabad, 500037, India.
Drug Deliv Transl Res. 2013 Dec;3(6):593-610. doi: 10.1007/s13346-013-0159-6.
The world is driving in to the era of transformation of chemical therapeutic molecules to biological genetic material therapeutics, and that is where the biological drugs especially "genes" come into existence. These genes worked as "magical bullets" to specifically silence faulty genes responsible for progression of diseases. Viral gene delivery research is far ahead of nonviral gene delivery technique. However, with more advancement in polymer science, new ways are opening for better and efficient nonviral gene delivery. But efficient delivery method is always considered as a bottleneck for gene delivery as success of which will decide the fate of gene in cells. During the past decade, it became evident that extracellular as well as intracellular barriers compromise the transfection efficiency of nonviral vectors. The challenge for gene therapy research is to pinpoint the rate-limiting steps in this complex process and implement strategies to overcome the biological physiochemical and metabolic barriers encountered during targeting. The synergy between studies that investigate the mechanism of breaking in and breaking out of nonviral gene delivery carrier through various extracellular and intracellular barriers with desired characteristics will enable the rational design of vehicles and revolutionize the treatment of various diseases.
世界正在进入将化学治疗分子转化为生物遗传物质治疗的时代,这就是生物药物特别是“基因”出现的地方。这些基因作为“神奇子弹”,专门沉默导致疾病进展的错误基因。病毒基因传递研究远远领先于非病毒基因传递技术。然而,随着聚合物科学的进步,为更好、更有效的非病毒基因传递开辟了新途径。但是,有效的传递方法一直被认为是基因传递的瓶颈,因为它的成功与否将决定基因在细胞中的命运。在过去的十年中,很明显,细胞外和细胞内的障碍都会影响非病毒载体的转染效率。基因治疗研究的挑战是确定这个复杂过程中的限速步骤,并实施克服靶向过程中遇到的生物物理化学和代谢障碍的策略。通过各种细胞外和细胞内障碍进行研究的机制,以及具有理想特性的非病毒基因传递载体的突破和突破的研究之间的协同作用,将能够实现载体的合理设计,并彻底改变各种疾病的治疗方法。