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Findings demonstrate that PTBP1 supports AML proliferation by fine-tuning the expression and splicing of AML-essential genes, providing a workflow for systematically annotating RNA-associated protein dependencies in cancer.
This study instigated a gene-specific targeted strategy by unlocking the regulatory network, and designed antisense oligonucleotides (ASOs) targeting important regulatory sites, demonstrating that ASO pharmacology is an actionable strategy for clinical development, challenging the existing paradigms in SF-mutated cance...
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