Triple-AAV intein-mediated gene therapy ameliorates dystrophic phenotype in MDC1A mice
29 July 2026 at 08:00
To overcome the strict packaging limits of AAV vectors, this study utilizes a triple-AAV system paired with orthogonal split inteins to reconstitute the exceptionally large LAMA2 protein. This scarless, multi-vector approach successfully rescues the dystrophic phenotype in vivo, offering a scalable platform for large-gene therapies.