Fields
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.
Details
- Avg Funding
- $1.2M
- Key Technologies
- Adeno-Associated Virus VectorsLipid NanoparticlesCRISPR Base EditorsAntisense OligonucleotidesLentiviral Vectors
- Subfields
- Viral Vector Gene DeliveryBase EditingPrime EditingCAR-T Cell TherapyRNA Therapeutics
- Top Institutions
- University of PennsylvaniaHarvard Broad InstituteNationwide Children's HospitalUCL Great Ormond StreetGénéthon France