Expert Reviews in Molecular Medicine: http://www.expertreviews.org/
Accession information: Vol. 8; Issue 1; 10 January 2006 Abstract
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Trans-splicing approach to repair mouse FVIII exon 16 mutation

Hengjun Chao and Christopher E. Walsh

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Figure 2. Trans-splicing approach to repair mouse FVIII exon 16 mutation. (a) A mouse FVIII PTM (coding for exons 16–26) binding to intron 15 of the mutant pre-mRNA of a haemophilia A mouse (FVIII-knockout mice were created by insertion of the neomycin resistance gene in exon 16 of the mouse gene for FVIII). (b) A trans-splicing event between the pre-mRNA transcribed from exon 15 of the endogenous mutant FVIII pre-mRNA and mRNA derived from exon 16 of the PTM generates a full-length repaired mouse FVIII mRNA. Abbreviations: BD, binding domain; BP, branchpoint; PTM, pre-trans-splicing molecule; Py, polypyrimidine tract; ss, splicing sequence.

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