Nukleinsyrebaserede

Nukleinsyrebaserede, also known as nucleic acid-based therapies, are a class of biopharmaceuticals that use nucleic acids, such as DNA, RNA, or their derivatives, to modulate gene expression or function. These therapies are designed to treat a wide range of diseases, including genetic disorders, infectious diseases, and cancer. The nucleic acids used in these therapies can be synthetic or derived from natural sources, and they can be administered in various forms, such as plasmids, oligonucleotides, or viral vectors.

The mechanism of action of nucleic acid-based therapies varies depending on the specific application. For example, in gene therapy, nucleic acids are used to introduce, modify, or silence specific genes to correct genetic defects or restore normal function. In RNA interference (RNAi) therapies, small interfering RNAs (siRNAs) or short hairpin RNAs (shRNAs) are used to silence specific genes by degrading their mRNA transcripts. In cancer therapies, nucleic acids are used to target and destroy cancer cells or inhibit their growth.

Nucleic acid-based therapies have shown promise in preclinical and clinical studies, with several therapies approved for use in humans. However, these therapies also face challenges, such as off-target effects, immune responses, and manufacturing and delivery issues. Ongoing research is focused on addressing these challenges and improving the efficacy and safety of nucleic acid-based therapies.