Bioinspired Nanoparticles for Gene Therapy

摘要

With the availability of human genome sequences and the advent of personalized medicine, gene therapy holds promise of treating diseases that are difficult to cure. But delivering nucleic acids as drugs or gene medicines has faced hurdles due to the lack of non-toxic delivery systems. With innovative peptide and lipid-based polymers as non viral vectors, scientists at the CSIR-Centre for Cellular and Molecular Biolog y (CCMB) have succeeded in delivering therapeutic genes and small interfering RNAs (siRNAs) into living cells effectively, using RNAi as a tool. RNAi is now recognized as a potential treatment strategy due to lack of cytotoxicity, minimal off-target transcriptional changes and non-immunogenicity. But, the lack of an efficient delivery system was until now the primary bottleneck for clinical application of siRNA.