A call to arms against ultra-rare diseases
Crooke, S.T.
Ultra-precision medicine
Crooke, S.T.
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.
