Therapeutic uses of Antisense molecules

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• Antisense molecules are special pieces of genetic material that help control protein production in the body. They work by attaching to specific genetic messages called messenger RNA (mRNA). These messages carry instructions for making proteins. Antisense molecules can block or break these messages, preventing harmful proteins from being produced. • Scientists have modified these molecules to make them more stable and effective inside the body. However, delivering them to the correct cells remains a challenge. • Antisense molecules are used to treat certain diseases, including genetic disorders and some other serious conditions. For example, nusinersen is used to treat spinal muscular atrophy, a genetic disease that affects muscle movement. • Despite their benefits, these treatments may cause side effects, be expensive, and have difficulty reaching the target cells. Researchers are working to improve their safety, effectiveness, and delivery methods. • In conclusion, antisense molecules are an important development in modern medicine. They offer a targeted way to treat diseases by controlling specific genetic messages. Further research may help make these treatments safer, more affordable, and accessible to more patients.

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