Deep screening of proximal and distal splicing-regulatory elements in a native sequence context

Yocelyn Recinos1, Dmytro Ustianenko1,2, Yow-Tyng Yeh1

  • 1Department of Systems Biology, Department of Biochemistry and Molecular Biophysics, Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA.

Summary

Researchers developed SpliceRUSH, a CRISPR-based screening tool, to map splicing-regulatory elements (SREs) in genes. This method identified novel SREs in the SMN2 gene, advancing RNA-based drug discovery for diseases like spinal muscular atrophy.

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