n-Lorem Foundation receives $500,000 donation for providing experimental antisense medicines to treat patients with ultra-rare diseases
CARLSBAD, Calif., April 14, [...]
Driving N-of-1 Therapies for Ultra-Rare Disease Patients
Antisense pioneer [...]
Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins
Migawa, M.T., Shen, W, Wan, W.B., Vasquez, G., Oestergaard, M.E., Low, A., De Hoyos, C.L., Gupta, R., Murray, S., Tanowitz, M., Bell, M., Nichols, J.G., Gaus, H., Liang, X.H., Swayze, E.E., Crooke, S.T., Seth, P.P.
Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index
Shen, W., De Hoyos, C.L., Migawa, M.T., Vickers, T.A., Sun, H., Low, A., Bell, T.A. 3rd., Rahdar, M., Mukhopadhyay, S., Hart, C.E., Bell, M., Riney, S., Murray, S.F., Greenlee, S., Crooke, R.M., Liang, X.H., Seth, P.P., Crooke, S.T.
Integrated Assessment of the Clinical Performance of GalNAc3-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience
Crooke, S.T., Baker B.F., Xia, S., Yu., R.Z., Viney, N.J., Wang, Y., Tsimikas, S., Geary, R.S. Download PDF
RNA-Targeted Therapeutics
Crooke, S.T., Witztum, J.L., Bennett, C.F., Baker, B.F.
