Addressing the Challenges of Treating Patients with Heterozygous Gain of Function Mutations
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.
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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