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dual-AAV.Var4 STRC
- Development stage
- Preclinical
- Lead developer
- Medizinische Hochschule Hannover
- Modality
- Gene Therapies
- Administration
- Otic
01
Overview
dual-AAV.Var4 STRC is a preclinical inner-ear gene-replacement therapy designed for autosomal recessive nonsyndromic hearing loss caused by pathogenic variants in the stereocilin gene. It uses the outer-hair-cell-tropic AAV capsid variant Var4 in a dual-AAV RNA-trans-splicing system, with each vector carrying a portion of the large STRC coding sequence. Co-delivery is intended to reconstitute full-length stereocilin expression in cochlear outer hair cells, restore stereociliary architecture, and improve auditory function. In mature Strc-deficient mice, the system produced persistent STRC expression, partial outer-hair-cell stereocilia rescue, and partial improvement in auditory brainstem response thresholds.
- Other names
- dual-AAV.Var4
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