Establishing a commercial solution for extremely rare genetic diseases
Crooke, S.T., Lo, A.W.
Splice-modulating antisense oligonucleotides targeting a pathogenic intronic variant in adult polyglucosan body disease correct mis-splicing and restore enzyme activity in patient cells
Thomas, R., Miyoshi, E., Akman, H.O., Storz, S.H.R., Goffena, J., Pytte, J., Miller, D.E., Skourti-Stathaki, K., Crooke, S.T.
A toxic gain-of-function variant in MAPK8IP3 provides insights into JIP3 cellular roles
Zhang, W., Mittal, S., Thomas, R., Foroughishafiei, A., Nunes Bastos, R., Chung, W., Skourti-Stathaki, K., Crooke, S.T.
Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder
Ziegler, A., Carroll, J., Bain, J.M., Sands, T.T., Fee, R.J., Uher. D., Kanner, C.H., Montes, J., Glass, S., Douville, J., Mignon, L., Gleeson, J.G., Crooke, S.T., Chung, W.K.
Safety and Tolerability of GalNAc3-Conjugated Antisense Drugs Compared to the Same-Sequence 2′-O-Methoxyethyl-Modified Antisense Drugs: Results from an Integrated Assessment of Phase 1 Clinical Trial Data
Baker B.F., Xia, S., Partridge, W., Engelhardt, J.A., Tsimikas, S., Crooke, S.T., Bhanot, S., Geary, R.S. Download PDF
A way forward for diagnosis of patients with extremely rare genetic mutations
Crooke, S.T., Kim-McManus, O.S., Dalby, K.
Personalized antisense oligonucleotides ‘for free, for life’ — the n-Lorem Foundation
Gleeson, J.G., Bennett, F.B., Carroll, J.B., Cole, T., Douville, J., Glass, S., Tekendo-Ngongang, C., Williford, A.C., Crooke, S.T.
Antisense Technology: A Review
Crooke, S.T., Liang, X.H., Baker, B.F., Crooke R.M.
Evaluating human mutation databases for ‘Treatability’ using personalized antisense oligonucleotides
Mittal, S., Tang, I., Gleeson, J.G.
