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Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?

25 Dec 2024

Abstract excerpt

INTRODUCTION: Duchenne muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle weakness and eventual death due to cardiomyopathy or respiratory complications. Currently, there is no cure for DMD, with standard treatments primarily focusing on symptom management. Using immunosuppressive measures and optimized vector designs allows for gene therapies to better address the genetic...

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PMID: 39720847
DOI: 10.1080/14740338.2024.2447072

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