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Oligonucleotides are chemically synthesized using building blocks, protected phosphoramidites of natural or chemically modified nucleosides or, to a lesser extent, of non-nucleosidic compounds. The oligonucleotide chain assembly proceeds in the 3' to 5' direction by following a routine procedure referred to as a "synthetic cycle".
Many oligonucleotide synthesis factories designed and manufactured their own custom platforms and little is known due to the designs being proprietary. The VLSS design continued to be refined and is continued in the QMaster synthesizer [105] which is a scaled down platform providing milligram to gram amounts of synthetic oligonucleotide.
In a similar manner to antibody therapeutics, Optimer therapeutics can be used as direct agonists or antagonists for the development of novel therapeutic moieties. [16] Additionally, Optimers can be used as conjugates for the targeted delivery of a range of drug cargo, such as chemotherapeutics , [ 14 ] gene silencing therapeutics, [ 22 ] and ...
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles. These therapeutics can be administered directly or through scaffold enrichment. [82] [83]
Synthetic oligonucleotides are used in various molecular biology applications, e.g., polymerase chain reaction (PCR), molecular beacons, microarrays, mutagenesis, RNAi, antisense and gene synthesis. Published bioinformatics algorithms can predict biophysical properties of oligonucleotides from their sequence and estimate oligonucleotide ...
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