RESEARCH FIELD

Gene Therapy

Gene therapy corrects genetic diseases by delivering functional copies of genes, editing faulty sequences, or silencing harmful gene expression using nucleic acid medicines and viral vectors. Adeno-Associated virus vectors have achieved durable therapeutic benefit in hemophilia, spinal muscular atrophy, and retinal dystrophies. CRISPR base editors make precise single-nucleotide changes without double-strand breaks, dramatically expanding the range of correctable mutations. mRNA lipid nanoparticle technology, validated at scale by COVID-19 vaccines, is being applied to metabolic and cardiovascular diseases. CAR-T cell therapies have transformed outcomes in certain blood cancers. Gene therapy is transitioning from rare disease niche to a platform for broad therapeutic application.