ABSTRACT

Traditional small molecule drugs are designed to interact with protein molecules that support or cause diseases. Protein drugs seek to replace the defective protein in cases where missing or defective protein is the cause of the disease. A gene is a sequence of the chromosome that codes for a specific protein. Thus, genes are made of DNA and contain information to produce specific proteins. Transcription is a nuclear process whereby information from DNA is transferred to messenger ribonucleic acid (mRNA). Antisense drugs inhibit the existing but abnormally expressed genes by blocking the transcription of DNA or the translation of mRNA. Overexpression of a particular protein can lead or contribute to a disease state, such as fibrosis and cancer. To create antisense drugs, nucleotides are linked together in short chains, called oligodeoxyribonucleotides (ODNs). When deoxyribonucleotides are linked in small chains, these are called oligodeoxyribonucleotides. The sequence of nucleotides in the antisense drugs is complementary to small segments of mRNA.