Aleksander Touznik

New developments in exon skipping and splice modulation therapies for neuromuscular diseases

T. Yokota, Aleksander Touznik, J. Lee, 2014, Expert opinion on biological therapy.

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

A. Nakamura, K. Nagaraju, Y. Aoki, 2015, Molecular therapy. Nucleic acids.

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

R. Maruyama, T. Yokota, Y. Echigoya, 2017, Scientific Reports.

Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy.

K. Mamchaoui, V. Mouly, S. Takeda, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

Allele-Selective Locked Nucleic Acid Gapmers for the Treatment of Fibrodysplasia Ossificans Progressiva Knock Down the Pathogenic ACVR1R206H Transcript and Inhibit Osteogenic Differentiation.

R. Maruyama, T. Yokota, Aleksander Touznik, 2022, Nucleic acid therapeutics.

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

R. Maruyama, T. Yokota, Y. Echigoya, 2017, Scientific Reports.

In Vitro Evaluation of Antisense-Mediated Exon Inclusion for Spinal Muscular Atrophy.

R. Maruyama, T. Yokota, Aleksander Touznik, 2018, Methods in molecular biology.