Concept of antisense molecules in medicinal chemistry
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Antisense molecules are synthetic segments of DNA or RNA, designed to mirror specific messenger RNA (mRNA) sequences and block protein production. One way to target the genetic material is to block mRNA by using “antisense DNA”, which prevents the message from ever becoming a protein. The use of antisense drugs to block abnormal disease-related proteins is referred to as antisense therapeutics. Synthetic short segments of DNA or RNA are referred to as oligonucleotides (ODNs). Whereas typical drugs target the proteins, it is possible, through antisense gene therapy, to target the genetic material itself before it is ever made into copies of harmful proteins. Antisense drugs have the promise to be more effective than conventional drugs, but one of the problems with antisense therapy is delivery. The efficacy of antisense ODNs is limited by the poor stability of the natural oligomers and the low efficacy of their cellular uptake. Nanotechnology has been used to improve this situation.
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Antisense molecules are synthetic segments of DNA or RNA, designed to mirror specific messenger RNA (mRNA) sequences and block protein production. One way to target the genetic material is to block mRNA by using “antisense DNA”, which prevents the message from ever becoming a protein. The use of antisense drugs to block abnormal disease-related proteins is referred to as antisense therapeutics. Synthetic short segments of DNA or RNA are referred to as oligonucleotides (ODNs). Whereas typical drugs target the proteins, it is possible, through antisense gene therapy, to target the genetic material itself before it is ever made into copies of harmful proteins. Antisense drugs have the promise to be more effective than conventional drugs, but one of the problems with antisense therapy is delivery. The efficacy of antisense ODNs is limited by the poor stability of the natural oligomers and the low efficacy of their cellular uptake. Nanotechnology has been used to improve this situation.
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