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AAV-NoSTOP suppressor-tRNA
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Intramuscular
01
Overview
AAV-NoSTOP suppressor-tRNA is an adeno-associated virus (AAV)-based gene therapy platform designed to treat genetic diseases caused by nonsense mutations. Developed by researchers at the University of Massachusetts Chan Medical School, the platform delivers engineered suppressor transfer RNA (sup-tRNA) genes that recognize and read through premature termination codons (PTCs). This mechanism allows the cellular translation machinery to produce full-length, functional proteins from mutated mRNAs. Additionally, the therapy works synergistically to inhibit nonsense-mediated mRNA decay (NMD), thereby increasing the stability and abundance of the target mRNA. The platform is disease-agnostic and has demonstrated preclinical efficacy in models of Hurler syndrome (MPS-I), CLN2 disease, and Duchenne muscular dystrophy (DMD) by targeting specific stop codons such as UAG, UGA, and UAA.
- Other names
- suppressor-tRNA platformsup-tRNA gene therapy
02
Targets
aaRS (Aminoacyl-tRNA synthetase family)PTC (Premature termination codon in mRNA within the ribosomal A site)NMD (Nonsense-mediated decay (NMD) machinery)
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