From Nucleotide to Gene Silencing

This book explores the revolutionary field of RNA-based therapeutics, focusing on the scientific journey from nucleotides to gene silencing through small interfering RNA (siRNA). It provides a comprehensive analysis of the mechanisms behind RNA interference, the structure-function relationship of siRNA molecules, and their therapeutic applications in modern medicine. Through detailed case studies of approved siRNA drugs such as Patisiran, Givosiran, and Inclisiran, the book highlights the translational path from laboratory discovery to clinical success. It also addresses formulation challenges, delivery systems, off-target effects, and future directions in precision medicine. Designed for researchers, clinicians, and biotechnology professionals, this work offers critical insights into the evolving landscape of gene-silencing technologies and their promise for treating genetic and metabolic disorders.