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A scalable and equitable framework for target and patient prioritisation in rare disease antisense therapeutics2026 medRxiv preprint Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access
Ella F. Whittle, Kylie Montgomery, Carme Camps, Nour M. Elkhateeb, Catherine Ryan, Sara Aguti, +26 more
1citations -
Opportunities and challenges for antisense oligonucleotide therapies2020 Journal of Inherited Metabolic Disease article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 1 patent
Elsa C. Kuijper, Atze J. Bergsma, W.W.M. Pim Pijnappel, Annemieke M Aartsma-Rus
155citations -
In Vivo Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy2025 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access
Anne-Fleur E. Schneider, Christa L. Tanganyika-de Winter, Hailiang Mei, Silvana M.G. Jirka, Xuyu Tan, Emily G. Thompson, +4 more
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Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy2025 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Sofia Stenler, Junyu Huang, Tirsa L.E. van Westering, Anna M. L. Coenen-Stass, Kaarel Krjutškov, Samir EL Andaloussi, +2 more
0citations