CORDIS Project
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This project investigates the epigenetic effects of gene therapy for spinal muscular atrophy. It aims to understand how gene delivery impacts the regulation of the SMN1 gene and its long-term effects on the epigenome in various tissues.
Spinal muscular atrophy (SMA) is a fatal neurodegenerative disorder in children, characterized by motor neuron loss and atrophy of muscle.
However, SMA affects many organ systems, since the causal gene, SMN1, is expressed in all cells.
Recently, an AAV9-mediated gene therapy, Zolgensma, was approved.
However, challenges remain.
To be effective, the systemically delivered AAV9- SMN1 needs to mimic the native regulation of the genomic SMN1.
In fact, recent findings demonstrated that delivery of AA…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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France, Paris
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
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