Article

In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach

2024-09-05

Abstract excerpt

Duchenne Muscular Dystrophy (DMD) is a pediatric disorder characterized by progressive muscle degeneration and premature death, with no cure at the moment. The currently most promising therapeutic avenue is based on gene replacement mediated by adeno-associated viruses (AAVs) using a shortened, but still functional, version of Dystrophin, known as microdystrophin (µDys), to fit AAV capacity. The limited improvemen...

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Literature Corpus work: b81e4c61-8681-5a4f-9402-b693120ce8df
DOI: 10.20944/preprints202409.0395.v1

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