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Abstract

SMaRT: A novel approach to gene therapy

Author(s): A Nayak, DV Kohli

Spliceosome-mediated RNA trans-splicing is a new technology for gene therapy that exploits the expressed genetic differences between normal and diseased cells. This technology may be applied to a wide range of diseases that involve the expression of unique or mutated genes. Spliceosome-mediated RNA trans-splicing technology can be employed to control the expression of any delivered gene by the presence of the chosen target pre-mRNA.

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