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AAV-TNNT1
- Development stage
- Preclinical
- Lead developer
- Clinic for Special Children
- Modality
- Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
AAV-TNNT1 is an adeno-associated virus (AAV) vector-based gene therapy designed to treat TNNT1-associated Nemaline Myopathy, also known as Amish Nemaline Myopathy (ANM). Developed by the University of Massachusetts Chan Medical School and the Clinic for Special Children, the therapy delivers a functional copy of the TNNT1 gene, which encodes the slow skeletal muscle isoform of troponin T. To prevent potential cardiotoxicity and off-target effects in fast-twitch myofibers, the vector incorporates microRNA target cassettes for de-targeting, restricting TNNT1 expression specifically to slow-twitch (type I) myofibers.
- Other names
- AAV-TNNT1 gene therapyAAV-TNNT-1 gene therapyAAV-TNNT 1 gene therapyAAV-TNNT1 vectorAAV-TNNT-1 vectorAAV-TNNT 1 vector
02
Targets
TNNT1 (Troponin T, slow skeletal muscle)
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