Oligonucleotides are (short) chains of (chemically modified) ribo- or deoxyribonucleotides. Their ability to bind to chromosomal DNA, mRNA, or non-coding RNA (ncRNA) through Watson-Crick and Hoogsteen base pairing offers possibilities for highly specific intervention in gene transcription, mRNA translation, gene repair, and recombination for therapeutic applications. In theory, a sequence of 15–17 bases occurs only once in the human genome, which would allow specific manipulation of single genes for oligonucleotides in this size range. In addition, therapeutic effects of oligonucleotides can be obtained through sequence-specific binding of transcription factors and intramolecular folding into structures that can bind to and interfere with the function of various biomolecules. Finally, cells display specific receptors for oligonucleotides. These receptors can activate a variety of immunological responses that can be of therapeutic value.
KeywordsCellular Uptake Antisense Oligonucleotide Peptide Nucleic Acid Hepatitis Delta Virus Nucleic Acid Molecule
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