126
Alternatively Spliced Genes
4.2
Potential Therapeutic Approaches
161
4.2.1
Oligonucleotide-based Approaches: Antisense, RNAi,
and Chimeric Molecules
161
4.2.2
Ribozymes
161
4.2.3
SMaRT
162
4.2.4
Chemical Compounds
162
5
Concluding Remarks
162
Acknowledgment
163
Bibliography
163
Books and Reviews
163
Primary Literature
166
Keywords
Pre-mRNA
Nascent transcripts that are precursors of mature messenger RNAs.
Exon
Gene regions that are present in mature messenger RNA transcripts.
Intron
Intervening sequences that are removed from pre-mRNA transcripts and not
represented in mature mRNA species.
Pre-mRNA Splicing
The process of removing introns from pre-mRNAs and ligating exons to form mature
mRNA transcripts.
UsnRNPs
Uridine-rich small nuclear ribonucleoprotein particles.
Spliceosome
The macromolecular machine in which pre-mRNA splicing reactions take place.
5
0
and 3
0
Splice Sites
Sequences that spliceosomes recognize at 5
0
and 3
0
ends of an intron. They are also
named splice donor and splice acceptor sites respectively.
Alternative Splicing
The process in which cells selectively use different combinations of splice sites to
generate two or more mRNA transcripts from a single pre-mRNA species.
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