Author
Ionis Pharmaceuticals (United States)ORCID 0000-0003-1430-5553Unclaimed ORCID profile · follow to get notified of new preprints
5 papers
Recent·Most cited
Mengnan Tian, Mehran Nikan, Miran Yoo…·Sungwon An
This study develops an siRNA-antibody conjugate targeting IGF1R for systemic and CNS delivery, showing effective knockdown in multiple tissues and good tolerability.
Ian J. Huggins, Michele Carrer, Jinro A. Santos…·Eric E. Swayze
This paper develops a VHH-Fc fusion protein to deliver siRNAs and ASOs across the BBB in rodents and NHPs, showing that binding affinity, DAR, and conjugation site affect delivery efficiency.
Mehran Nikan, Thazha P. Prakash, Guillermo Vasquez…·Eric E. Swayze
This paper introduces modified nucleosides in siRNA seed regions to enhance specificity and reduce off-target effects while maintaining on-target activity.
Jacqueline G. O’Rourke, Gemma Bachmann, Curt Mazur…·Holly B. Kordasiewicz
This study characterizes an acute sedation response caused by phosphorothioate ASOs after CNS delivery, showing it is sequence-, dose-, and chemistry-dependent and can be mitigated.
Brendon E. Cook, Thomas C. Pickel, Sangram Nag…·Maciej A. Kaliszczak
This paper demonstrates a pretargeted PET imaging method to visualize antisense oligonucleotide distribution in non-human primate brains after intrathecal delivery.