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Cristina Fernández-Avilés

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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. · 0 citations

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