LIST OF FIGURES
Figure page
1. Trans-acting ribozymes can be designed to specifically cleave an mRNA
substrate........................................................................................................ 4
2. The secondary structure of self-splicing introns ..................................................7
3. The design of a trans-splicing group I intron for gene therapy applications ........9 4. The splicing mechanism of the group II intron..................................................11
5. Cleavage of the tRNA 5' leader sequence by RNase P......................................13
6. An RNA guide sequence allows the cleavage of a specific mRNA sequence by
RN ase P................................................ ...................................................... 14
7. Self-cleaving ribozymes resolve concatemers formed by rolling-circular
replication into individual genomic molecules............................................. 17
8. The structure of the trans-cleaving HDV ribozyme ..........................................18
9. The structure of the trans-cleaving hammerhead ribozyme................................19
10. The structure of the trans-cleaving hairpin ribozyme.........................................21
11. Structure of the rod photoreceptor cell ..............................................................26
12. Rod cells are intimately associated with RPE cells............................................27
13. Structure of the rod opsin protein......................................................................28
14. The visual transduction cascade .......................................................................29
15. Retinitis Pigmentosa results in a gradual loss of peripheral vision.....................31
16. The G90D mutation disrupts a salt bridge required for the normal structure of the
rod opsin protein......................................... ................................................ 34
17. The nucleotide and amino acid sequence of the transgenes used to create the
m ouse m odels for this project...................................................................................37
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