Targeted drugs aim to pinpoint the exact location in the body where diseased tissue is located and where the medicine is required. The manifold benefits of administering a targeted drug include heightened efficacy, as the drug is meticulously designed for specificity, thereby reducing side effects, and minimizing damage to healthy tissue. Consequently, this approach enhances the patient’s quality of life during treatment.
Oligonucleotides (ONs), specifically designed short chains of DNA or RNA, have emerged as a crucial tool with immense potential in personalized medicine. These therapeutic ONs are already in use for conditions, such as certain types of muscular dystrophy and liver disease, which conventional drugs cannot address.
Depending on the type, ONs can function by, preventing or changing the production of a protein in the cell, particularly beneficial in diseases caused by the overproduction of a specific protein.
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