Details

Autor(en) / Beteiligte

• Puttaraju, M
• DiPasquale, Janet
• Baker, Carl C
• Mitchell, Lloyd G
• Garcia-Blanco, Mariano A

Titel

Messenger RNA Repair and Restoration of Protein Function by Spliceosome-Mediated RNA Trans-Splicing

Ist Teil von

• Molecular therapy, 2001-08, Vol.4 (2), p.105-114

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2001

Quelle

MEDLINE

Beschreibungen/Notizen

• 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.