Antisense oligonucleotide as a therapeutic approach in amyotrophic lateral sclerosis
Korobeynikov, V.A., Lyashchenko, A.K., Blanco-Redondo, B., Jafar-Nejad, P. Shneider, N.A.
IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Drug Evaluation and Research (CDER)
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Perinuclear positioning of endosomes can affect PS-ASO activities
Liang, X.H., Nichols, J.G., Tejera, D., Crooke, S.T.
IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations
U.S. Department of Health and Human Services
Food and Drug Administration
Center for Drug Evaluation and Research (CDER)
Download PDF
Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides
Anderson, B.A., Freestone, G.C., Low, A., De-Hoyos, C.L., Iii, W.J.D., Østergaard, M.E., Migawa, M.T., Fazio, M., Wan, W.B., Berdeja, A., Scandalis, E., Burel, S.A., Vickers, T.A., Crooke, S.T., Swayze, E.E., Liang, X., Seth, P.P.
Towards Next Generation Antisense Oligonucleotides: Mesylphosphoramidate Modification Improves Therapeutic Index and Duration of Effect of Gapmer ASO
Anderson, B.A., Freestone, G.C., Low, A., De-Hoyos, C.L., Iii, W.J.D., Østergaard, M.E., Migawa, M.T., Fazio, M., Wan, W.B., Berdeja, A., Scandalis, E., Burel, S.A., Vickers, T.A., Crooke, S.T., Swayze, E.E., Liang, X., Seth, P.P.
A call to arms against ultra-rare diseases
Crooke, S.T.
