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Updated: Jul 28, 2026

Single-step Purification of Macromolecular Complexes Using RNA Attached to Biotin and a Photo-cleavable Linker
Published on: January 3, 2019
Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing
M Puttaraju1, J DiPasquale, C C Baker
1Intronn LLC, 840 Main Campus Drive, Raleigh, North Carolina 27606, USA.
Abstract:
The functional repertoire of the human genome is amplified by the differential assortment of exons. Spliceosome-mediated RNA trans-splicing can mobilize these packets of genetic information to reprogram mRNAs. In principle, this process could repair defective transcripts in loss-of-function genetic disorders in humans. We developed a tractable lacZ repair system to serve as a model for these genetic disorders. Targeted pre-trans-splicing RNA molecules efficiently and specifically repaired mutated lacZ transcripts and restored enzymatic activity in human cells. The development of this model confirms the potential for spliceosome-mediated RNA trans-splicing in genetic repairs and provides a powerful tool for rational design and in vitro evolution of pre-trans-splicing molecules.
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