RNA Processing and Role of Alternative Splicing on Diseases and its Therapeutic Uses

RNA processing or splicing is a method by which mRNA is produced from pre mRNA which becomes ready for translation into protein. But there is another very important process of RNA processing in eukaryotes that is called Alternative Splicing. This process is very important in these organisms particularly in human being. It is known that human has 20,000 genes but this not sufficient for such complexity and diversity in human as it was known that one gene gives one protein. There is another important process called Alternative processing by which one gene can produce more than one protein. Splicosome machinery important for RNA processing has been discussed with methods ofTrans-splicing and Alternative splicing. Any defect or dysregulation in Spliceosome and alternative processing has been found to be the main cause of many rare diseases like neurogenerative and Cancer. The origin of these diseases due to dysregulation in alternative splicing has been discussed in detail. Therapeutic uses of Antisense oligonucleotides, small molecules and small molecule modulators have been discussed. The mechanism of action of Antisense oligonucleotides used for the correction of defects in RNA processing is also discussed.