Antisense oligonucleotide. An antisense oligonucleotide (ASO) is a single-stranded, synthetic RNA (or DNA) sequence. ASOs are designed to selectively bind via complementary base-pairing to messenger RNA (mRNA) and are the basis for one type of RNA-based therapeutics being explored to treat cancer and genetic disorders. RNA-based therapeutics The molecules tested for antisense activity, which contained only LNA and DNA nucleotides, seem to offer an attractive set of properties when compared with many other antisense oligonucleotides: (i) biological stability, (ii) RNase H activation, (iii) lack of detectable toxicity, and (iv) potent biological activities. The present LNA-based DNA normally has two strands, i.e., the sense strand and the antisense strand. In double-stranded DNA, only one strand codes for the mRNA which, in turn, is translated into protein. This template DNA strand is referred to as the antisense strand. The strand that does not code for RNA is called the sense strand/coding strand.The goal of the antisense approach is the downregulation of a molecular target, usually achieved by induction of RNase H endonuclease activity that cleaves the RNA-DNA heteroduplex with a significant reduction of the target gene translation (Figure 1).
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Antisense inhibition of gene expression relies primarily on the simple rules of base pairing of nucleic acids (see Fig. 1 ). When a gene is transcribed, the resulting messenger RNA (mRNA) contains
b) only one of the two strands of DNA possesses correct hereditary information. It is known as sense strand. Its complementary strand is called antisense strand. Antisense RNA that is made from the DNA strand that is complementary to the sense stand of the DNA.
Antisense oligonucleotides are designed to hybridize specific ing in DNA/RNA heteroduplex structures formed by antisense drugs and their targets is a new arearnhzq.
