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Strong Ubiquitous Mini-Promoters for AAV-Based Gene Therapy
TS-004616 — A method for targeted delivery and constitutive expression of large genes using adeno-associated viral (AAVs) vectors that contain ubiquitous mini-promoters. Incorporation of the compact mini-promoters will increase the cargo space of the AAVs such that larger genes can be packaged into the vectors.
Adeno-associated viral (AAV) vectors are highly efficacious gene therapy vectors are safe and highly efficacious for gene therapy and have advanced to clinical trials for a number of disorders. However, AAV vectors have a limited cargo capacity (4.7 kilobase pairs), thus over 20% of human genes are …
- College:
- Inventors: Hester, Mark; Moser, Morgan
- Licensing Officer: Zalucha, Ellen
