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Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides.
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- International Journal of Molecular Sciences, 2019, v. 20, n. 20, p. 5030, doi. 10.3390/ijms20205030
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- Article
Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies.
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- Biomedicines, 2021, v. 9, n. 5, p. 552, doi. 10.3390/biomedicines9050552
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- Article
Single Stranded Fully Modified-Phosphorothioate Oligonucleotides can Induce Structured Nuclear Inclusions, Alter Nuclear Protein Localization and Disturb the Transcriptome In Vitro.
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- Frontiers in Genetics, 2022, v. 13, p. 1, doi. 10.3389/fgene.2022.791416
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- Article
ALS Genetics, Mechanisms, and Therapeutics: Where Are We Now?
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- Frontiers in Neuroscience, 2019, v. 13, p. 1, doi. 10.3389/fnins.2019.01310
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- Article
A Precision Therapy Approach for Retinitis Pigmentosa 11 Using Splice-Switching Antisense Oligonucleotides to Restore the Open Reading Frame of PRPF31.
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- International Journal of Molecular Sciences, 2024, v. 25, n. 6, p. 3391, doi. 10.3390/ijms25063391
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- Article