University of Florida Research Foundation; Incorporated
发明人:
Jacqueline A. Hobbs,Scott A. Rivkees,Arun Srivastava,Laura A. Small
申请号：
US15741253
公开号：
US20180193489A1
申请日:
2016.07.01
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Provided herein are nucleic acids, recombinant adeno-associated virus (rAAV) particles, and compositions, as well as methods of use thereof for transducing medullary thyroid carcinoma cells and in treatment of disease, such as medullary thyroid carcinoma. In some aspects, the nucleic acid comprises a truncated calcitonin promoter, which is optionally encapsidated within a rAAV particle. In other aspects, the rAAV particle is a rAAV particle having a mutation in a surface-exposed amino acid, such as tyrosine, threonine, or serine, that enhances transduction of the particle into medullary thyroid carcinoma cells.
