CORDIS Project
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MYOCURE aims to create a gene therapy platform targeting rare muscle disorders, particularly myotubular myopathy and glycogen storage disease type II. The project focuses on enhancing gene transfer and expression while minimizing immune reactions, with plans for preclinical validation and eventual clinical trials.
The objective of MYOCURE is to develop an innovative gene therapy platform to cure rare hereditary muscle disorders, specifically focusing on myotubular myopathy (MTM) and glycogen storage disorder (GSD) type II.
These are attractive diseases for gene therapy since they compromise a diverse family of rare genetic diseases typically caused by single gene defects that often provoke significant morbidity and mortality due to skeletal muscle, cardiac and/or diaphragm dysfunction.
There are no effect…
VRIJE UNIVERSITEIT BRUSSEL
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France, Paris
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Germany, Heidelberg
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Spain, Cerdanyola Del Valles
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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