抽象的

Decoy oligonucleotides: silencing the negativity in gene therapy?

Nicole Nogoy

Throughout the last 15 years, the concept of replacing deficient genes or knocking down the expression of genes that cause disease has been consistently developed and is under scrutiny by the public. This concept is known as gene therapy. Several strategies of gene therapy have been developed, ranging from the use of retroviral vectors carrying a deficient gene of interest whose aim is to re-insert the deficient gene into the patient genome, to the use of DNA technology to inhibit target gene expression. In particular, the application of DNA technology, such as RNA interference and antisense strategies, to silence or regulate transcription of disease-related genes in vivo has important therapeutic potential. This perspective highlights both the positive and negative aspects of gene therapy and raises questions as to how effective the use of retroviral vectors and decoy oligonucleotides can be as gene-therapy strategies.