Thazha P. Prakash

This study develops an siRNA-antibody conjugate targeting IGF1R for systemic and CNS delivery, showing effective knockdown in multiple tissues and good tolerability.

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.

This paper introduces NMA-modified antisense oligonucleotides that show improved potency compared to MOE-modified SSOs in modulating splicing for SMA and Dravet syndrome.

This paper introduces modified nucleosides in siRNA seed regions to enhance specificity and reduce off-target effects while maintaining on-target activity.

This study identifies SIRT2 as a regulator of β-cell proliferation, showing its role in controlling β-cell expansion during hyperglycemia while preserving feedback mechanisms.