Gene-based medicine is transforming modern healthcare by offering precise, personalized interventions that target the genetic causes of disease. Breakthroughs in gene editing technologies, including clustered regularly interspaced short palindromic repeat (CRISPR)-associated (Cas) nuclease technologies (CRISPR-Cas9), base editing, and prime editing, are enabling promising therapeutic applications for rare inherited disorders and complex conditions like cancer. Furthermore, improvements in both viral and non-viral delivery methods are expanding clinical possibilities and enhancing safety measures. Despite these advancements, challenges such as off-target effects, ethical considerations, production complexities, and high costs continue to hinder widespread adoption. This review explores current innovations in gene-based medicine, addresses remaining obstacles, and outlines future directions, emphasizing the transformative potential of genomic-driven therapies for patients worldwide.
Ralf Weiskirchen (Mon,) studied this question.