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AAV.U7snRNA VES exons 6-8
- Development stage
- Preclinical
- Lead developer
- Nationwide Children's Hospital
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
- Administration
- Intramuscular, Intravenous
01
Overview
AAV.U7snRNA VES exons 6-8 is an experimental gene therapy designed for the treatment of Duchenne muscular dystrophy (DMD) in patients with mutations within exons 6, 7, and 8. Developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, this vectorized exon skipping (VES) approach utilizes an adeno-associated virus (AAV) to deliver a U7 small nuclear RNA (U7snRNA) fused with antisense sequences. The therapy aims to induce the simultaneous skipping of exons 6, 7, and 8 in the DMD pre-mRNA, thereby restoring the reading frame and allowing for the production of a truncated but functional dystrophin protein. This strategy is intended to provide a permanent therapeutic effect with a single systemic administration, overcoming the limitations of traditional antisense oligonucleotides (ASOs) that require repeated dosing.
- Other names
- vectorized exon skipping exons 6-8
02
Targets
Dystrophin pre-mRNA exon 6, 7, and 8 splice regulatory sequencesHNRNPA1 (Heterogeneous nuclear ribonucleoprotein A1)
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