期刊
BRIEFINGS IN FUNCTIONAL GENOMICS
卷 10, 期 3, 页码 151-164出版社
OXFORD UNIV PRESS
DOI: 10.1093/bfgp/elr020
关键词
RNA splicing; alternative splicing; splicing mutations; antisense oligonucleotides; splice correction; DMD
资金
- National Institute for Health Research [ACF-2007-26-002] Funding Source: researchfish
The majority of human genes that encode proteins undergo alternative pre-mRNA splicing and mutations that affect splicing are more prevalent than previously thought. The mechanism of pre-mRNA splicing is highly complex, requiring multiple interactions between pre-mRNA, small nuclear ribonucleoproteins and splicing factor proteins. Regulation of this process is even more complicated, relying on loosely defined cis-acting regulatory sequence elements, trans-acting protein factors and cellular responses to varying environmental conditions. Many different human diseases can be caused by errors in RNA splicing or its regulation. Targeting aberrant RNA provides an opportunity to correct faulty splicing and potentially treat numerous genetic disorders. Antisense oligonucleotide therapies show particular promise in this area and, if coupled with improved delivery strategies, could open the door to a multitude of novel personalized therapies.
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