AAV9-troponin gene therapy

**AAV9-troponin gene therapy** is a non-proprietary preclinical AAV9-mediated skeletal-muscle gene-replacement approach being investigated for **TNNT1 nemaline rod myopathy**. The reported experimental vectors deliver the three interacting troponin genes—**TNNC1**, **TNNI1**, and **TNNT1**—to restore expression of slow skeletal-muscle troponin-complex proteins. In a TNNT1-knockout mouse model, intramuscular administration of a dual-cassette AAV9 construct produced low but detectable troponin T expression, whereas a single-cassette construct did not produce detectable troponin T. The term does not identify one uniquely named drug candidate because it encompasses more than one experimental AAV9 vector design.

Other names

AAV9-troponin gene therapyAAV-9-troponin gene therapyAAV 9-troponin gene therapy