Author
Leiden University Medical CenterORCID 0009-0004-5025-0830Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
Recent·Most cited
Maaike van Putten, Margot M. L. Linssen, C. Tanganyika-de Winter…·Peter Hohenstein
The paper develops four humanized DMD mouse models with specific human exon deletions to test exon skipping therapies for Duchenne muscular dystrophy.
Paola Mantuano, Antonietta Mele, Brigida Boccanegra…·Maaike van Putten
This study compares the natural history of two DMD mouse models, BL10-mdx and D2-mdx, across two sites, finding distinct phenotypes and highlighting the importance of genetic background.
Enyang Han, Katherine Webster, Tiberiu Loredan Stan…·Tom A. Hartl
The paper identifies sequence and chemical features of ASOs that efficiently induce exon 44 skipping in DMD and defines design principles for therapeutic ASOs.