Author
Ionis Pharmaceuticals (United States)ORCID 0000-0002-7885-6525Unclaimed ORCID profile · follow to get notified of new preprints
Mutual co-authors in cluster
3 papers
Recent·Most cited
Karen Ling, Thazha P. Prakash, Jinghua Yu…·Frank Rigo
This paper introduces NMA-modified antisense oligonucleotides that show improved potency compared to MOE-modified SSOs in modulating splicing for SMA and Dravet syndrome.
Gerarda Cappuccio, Guantong Qi, Xuan Qin…·Mirjana Maletić‐Savatić
The paper identifies a disrupted mitochondrial-purine metabolism link in MECP2 Duplication Syndrome across multiple models, linking it to neurodevelopmental issues.
Matthew P. Stratton, Jessica L. Centa, Vicki J. Swier…·Michelle L. Hastings
A splice-switching antisense oligonucleotide delivered via intravitreal injection mitigates early retinal dysfunction in a pig model of CLN3 Batten disease.