Gene Therapy & CRISPR Research
AI-powered research into CRISPR gene editing, CAR-T cell engineering, antisense oligonucleotides, and RNA therapeutics across neurological and oncological applications.
Key Mechanisms
- CRISPR-Cas9 gene editing and base editing
- Antisense oligonucleotide (ASO) gene silencing
- AAV vector-mediated gene delivery
- CAR-T cell engineering for solid tumors
Notable Compounds
- Tofersen (SOD1 ASO)
- CRISPR-Cas9 constructs
- AAV9 vectors
- CAR-T constructs