Arthur H M Burghes

5PUBLICATIONS
38CO-AUTHORS
Inorganic materials (incl. nanomaterials)Neurology and neuromuscular diseasesRegenerative medicine (incl. stem cells)Gene and molecular therapy
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Publications (5)

|Aug 18, 2025
Sm-site containing mRNAs can accept Sm-rings and are downregulated in Spinal Muscular Atrophy.

Anton J Blatnik, Manu Sanjeev, Jacob Slivka

|Mar 20, 2024
The ClC-1 chloride channel inhibitor NMD670 improves skeletal muscle function in rat models and patients with myasthenia gravis.

Martin Skov, Titia Q Ruijs, Thomas S Grønnebæk

|Mar 30, 2023
Base editing rescue of spinal muscular atrophy in cells and in mice.

Mandana Arbab, Zaneta Matuszek, Kaitlyn M Kray

|Oct 05, 2021
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue.

Gretchen Thomsen, Arthur H M Burghes, Caroline Hsieh

|Aug 27, 2021
What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective.

Anton J Blatnik, Vicki L McGovern, Arthur H M Burghes

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