# 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.

*Source: [https://selltoscientists.com/fields/gene-therapy/](https://selltoscientists.com/fields/gene-therapy/)*

**Researcher count:** 31,000
**Average funding:** $1.2M

## Subfields

- Viral Vector Gene Delivery
- Base Editing
- Prime Editing
- CAR-T Cell Therapy
- RNA Therapeutics

## Key technologies

- Adeno-Associated Virus Vectors
- Lipid Nanoparticles
- CRISPR Base Editors
- Antisense Oligonucleotides
- Lentiviral Vectors

## Top institutions

- University of Pennsylvania
- Harvard Broad Institute
- Nationwide Children's Hospital
- UCL Great Ormond Street
- Généthon France

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