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Published in 2018 at "Biochemical and biophysical research communications"
DOI: 10.1016/j.bbrc.2018.02.160
Abstract: Alternative splicing (AS) generates multiple isoforms from a single precursor mRNA, and these isoforms usually exhibit different tissue distributions and functions. Aberrant protein isoforms can lead to abnormalities in protein function and may even result…
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Keywords:
tak1;
switching oligonucleotides;
splice switching;
alternative splicing ... See more keywords
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Published in 2023 at "RNA"
DOI: 10.1261/rna.079540.122
Abstract: Splice-modulating antisense oligonucleotides (ASOs) offer treatment options for rare neurological diseases, including those with very rare mutations, where patient-specific, individualized ASOs have to be developed. Inspired by the development of milasen, the 1 Mutation 1…
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Keywords:
splice switching;
splice;
development tailored;
development ... See more keywords