Author

Nina Raben

National Institutes of HealthORCID 0000-0001-9519-3535Unclaimed ORCID profile · follow to get notified of new preprints

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Molecular Biology· 1

Signature Assays, Organisms & Methods

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Approach:experimental assay· 1 Organism:mouse· 1

Frequent Co-Authors

Alyaa ShmaraiD· 1 shared Angela MartiniD· 1 shared Cheng ChengiD· 1 shared Hong Zhen YiniD· 1 shared Howard Yu· 1 shared John H. WeissiD· 1 shared Jonathan LeeiD· 1 shared Lac TaiD· 1 shared

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2025-2025 · peak 1/yr

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1 shared paperMolecular Biology

Alyaa ShmaraORCID

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1 paper

Recent·Most cited

Molecular BiologyClinical and Translational MedicinePeer-reviewedApr 1, 2025

Skeletal muscle effects of antisense oligonucleotides targeting glycogen synthase 1 in a mouse model of Pompe disease

Lan Nguyen Weiss, Michele Carrer, Alyaa Shmara…·Virginia E. Kimonis

This study shows that ASO-mediated Gys1 knockdown in a mouse model of Pompe disease reduces glycogen accumulation and improves motor function, both alone and in combination with ERT.

4 citations

mouseexperimental assay

𝕏 PostBluesky