New Research on Gene Therapy and Lafora Disease

Researchers present gene replacement therapy as an approach for Lafora disease in the recently published article “Malin restoration as proof of concept for gene therapy for Lafora disease.” 

This paper shows that restoring malin expression in a mouse model that lacks it can reduce Lafora Body accumulation and neuroinflammation. 

This proof-of-concept suggests that it would be beneficial to develop gene therapy for restoring malin expression in patients where malin is mutated or missing.

If you have questions about gene therapy and Lafora Disease, please feel free to contact our Science Director, Dr. Kit Donohue, at  

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