This page discusses antisense oligonucleotides, synthetic polymers designed to inhibit protein synthesis by binding to mRNA. They work by blocking ribosome function, degrading mRNA, or preventing spli...This page discusses antisense oligonucleotides, synthetic polymers designed to inhibit protein synthesis by binding to mRNA. They work by blocking ribosome function, degrading mRNA, or preventing splicing errors. Effective therapy requires evasion of nucleases and minimal side effects, often achieved through chemical modifications and targeted delivery.
This page outlines the drug testing process, which starts with testing numerous chemicals on lab animals and may lead to human trials through an IND application to the FDA. The human trials consist of...This page outlines the drug testing process, which starts with testing numerous chemicals on lab animals and may lead to human trials through an IND application to the FDA. The human trials consist of three phases: Phase I focuses on safety in healthy volunteers, Phase II tests efficacy and side effects in patients, and Phase III collects comprehensive safety and effectiveness data. Upon successful completion, a NDA is submitted, and the drug is branded for market release.