Jul 2026
Triple AAV Intein-Mediated Gene Therapy Ameliorates Dystrophic Phenotype in MDC1A Mice.
Using a combination of three AAVs, each encoding for one fragment of the laminin-α2 protein flanked by short split intein resulted in the complete reconstitution of the Laminin-α2 and an improvement of the histopathological features of the dy2j dystrophic mouse model.
Núria Rafel-Millan, Carles Bayod-Girón, Maria Pallarès-Masmitjà et al.
· Molecular Therapy · 0 citations