Computer aid design of antisense oligonucleotide in gene therapy--review / 中国实验血液学杂志

ABSTRACT

In this paper, the situation on antisense oligonucleotide as a means of gene therapy was outlined, and the main factors impeding its progress at present was summarized. The one of main factors is the efficiency of antisense oligonucleotide as a drug and the other is the side-effect in clinical use. At the level of cell and gene, these influential factors were analyzed in detail. The main factor that makes side-effect in using antisense oligonucleotide is the difficulty to distinguish effectively homologous-gene from target gene. The another factor is the secondary structure and three-dimensional structure of target gene that seriously affect antisense oligonucleotide to arrive at target position. The third problem is what can affect antisense oligonucleotide transmission and quick annealing. How use computer technique to analyze fully the target gene of antisense oligonucleotide including the secondary structure and homology of target gene, and to design effective antisense oligonucleotide, in order to reduce its side-effect in clinical use of antisense oligonucleotide as a drug of gene therapy, and the computer-aid design method were described.