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Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing Sazani P; Kole RJ Clin Invest 2003[Aug]; 112 (4): 481-6An estimated 60% of all human genes undergo alternative splicing, a highly regulated process that produces splice variants with different functions. Such variants have been linked to a variety of cancers, and genetic diseases such as thalassemia and cystic fibrosis. This Perspective describes a promising approach to RNA repair based on the use of antisense oligonucleotides to modulate alternative splicing and engender the production of therapeutic gene products.|*Alternative Splicing[MESH]|*Antigens, Surface[MESH]|Carboxypeptidases/genetics[MESH]|Cystic Fibrosis Transmembrane Conductance Regulator/genetics[MESH]|Dystrophin/genetics[MESH]|Globins/genetics[MESH]|Glutamate Carboxypeptidase II[MESH]|Humans[MESH]|Ligands[MESH]|Models, Biological[MESH]|Models, Genetic[MESH]|Oligonucleotides, Antisense/*therapeutic use[MESH]|Polycystic Kidney Diseases/genetics[MESH]|Proto-Oncogene Proteins c-bcl-2/genetics[MESH]|Proto-Oncogene Proteins c-myc/genetics[MESH]|RNA, Messenger/metabolism[MESH]|Receptor, Insulin/genetics[MESH]|Receptors, GABA-A/genetics[MESH]|Receptors, Interleukin-5[MESH]|Receptors, Interleukin/genetics[MESH]|bcl-X Protein[MESH]|tau Proteins/genetics[MESH] |
