1. Entering the Era of Precision Medicine to Treat ALS.
2. Allele-Selective Thiomorpholino Antisense Oligonucleotides as a Therapeutic Approach for Fused-in-Sarcoma Amyotrophic Lateral Sclerosis.
3. Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro.
4. Translational development of splice-modifying antisense oligomers.
5. ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?
6. Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies.
7. Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides.
8. Structural Variants May Be a Source of Missing Heritability in sALS.
9. Synucleinopathy in Amyotrophic Lateral Sclerosis: A Potential Avenue for Antisense Therapeutics?
10. p62 overexpression induces TDP-43 cytoplasmic mislocalisation, aggregation and cleavage and neuronal death.
11. Novel STMN2 variant linked to amyotrophic lateral sclerosis risk and clinical phenotype.
12. Intronic NEFH variant is associated with reduced risk for sporadic ALS and later age of disease onset.
13. Short structural variants as informative genetic markers for ALS disease risk and progression.
14. New perspectives on cytoskeletal dysregulation and mitochondrial mislocalization in amyotrophic lateral sclerosis.
15. Disease-modifying effects of an SCAF4 structural variant in a predominantly SOD1 ALS cohort.
16. Association of a structural variant within the SQSTM1 gene with amyotrophic lateral sclerosis.
17. Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies.
18. In vitro validation of phosphorodiamidate morpholino oligomers.
19. Antisense-mediated splice intervention to treat human disease: the odyssey continues.
20. Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN Transcript.
21. Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy.
22. A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice.
23. Targeted Molecular Therapeutics for Parkinson's Disease: A Role for Antisense Oligonucleotides?
24. A Splice Intervention Therapy for Autosomal Recessive Juvenile Parkinson’s Disease Arising from Parkin Mutations.
25. Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping.
26. Antisense oligonucleotides and their applications in rare neurological diseases.
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