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Schlagwörter:
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Zusammenfassung:
The complex compositional and conformational dynamics of spliceosomes required for regulated splicing are prone to malfunction when mutations affect splicing factors or cis-acting regulatory sequences. Indeed, many such mutations have been linked to heritable diseases or malignancies in humans. Small molecule modulators and antisense oligonucleotides or analogs harbor great potential for therapies and several substances that can modulate splicing events have entered clinical trials.
