How does the medicine work? 

Specific genes contain information to produce specific proteins. The information required for the human body to produce all proteins is contained in the genome and its collection of more than 1,00,000.00 genes. Genes are made up of deoxyribonucleic acid (DNA). In DNA, the nucleotides Adenine, Thymine, Guanine, Cytosine are weakly bound or paired by hydrogen bonds to complementary nucleotides on the other hand A to T,G to C. Such highly specific complementary base pairing is the essence of information transfer from DNA to its intermediary messenger RNA (mRNA), and carries the information, spelled out by the specific sequences of bases necessary for the cell to produce a specific protein. During transcription of information from DNA into mRNA, the two complementary strands of the DNA partly uncoil. The DNA is used as a template for transcribing enzymes which assemble mRNA--a process called transcription; mRNA migrates into the cell where other cellular structures called ribosome read the encoded information, its mRNA base sequence, and in so doing ,string together amino acids to form specific protein. This process is called translation.

The medicines are complementary strands of small segments of mRNA. The medicines, nucleotides are linked together in short chains called oligonucleotides. The medicine is designed to bind to a specific sequence of nucleotides in its mRNA target to inhibit" production of the protein encoded by the target mRNA.. By acting at this earlier stage in the disease-causing process to prevent the production of a disease-causing protein, the medicines have the potential to provide greater therapeutic benefit than traditional medicines which do pot act until the disease- causing protein has already been produced. The medicines have the potential to be much more selective than traditional medicines and therefore more effective, because they bind to mRNA targets at multiple points of interaction at a single receptor site. Traditional medicines usually bind at only two points of interaction.

The design of the medicines has the advantage of being less complex, more rapid and more efficient. Rational medicines' design usually begins by characterizing the three-dimensional structure of the protein target in order to design a prototype medicine to interact with the target. Proteins, however, are complex molecules whose structure is difficult to predict. In contrast, the compounds are designed to bind to mRNA whose structures are more easily understood and predicted.

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