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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]
In protein synthesis, DNA is first transcribed into mRNA, and then translated in an amino acid sequence. Gapmers take advantage of this biological pathway by binding to the mRNA target. [7] In humans, the gapmer DNA-mRNA duplex is degraded by RNase H1. [1] [5] The degradation of the mRNA prevents protein synthesis [7] [8] Gapmer Mechanism of Action
Oligonucleotide synthesis is the chemical synthesis of relatively short fragments of nucleic acids with defined chemical structure ().The technique is extremely useful in current laboratory practice because it provides a rapid and inexpensive access to custom-made oligonucleotides of the desired sequence.
An Escherichia coli-based open cell-free synthesis (OCFS) allows the synthesis of proteins containing site-specifically incorporated non-natural amino acids and has been optimized for predictable high-yield protein synthesis and folding. The absence of a cell wall allows the addition of non-natural factors to the system to manipulate ...
Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics.Commonly made in the laboratory by solid-phase chemical synthesis, [1] these small fragments of nucleic acids can be manufactured as single-stranded molecules with any user-specified sequence, and so are vital for artificial gene synthesis, polymerase ...
Combination of exceptional targeting capabilities of monoclonal antibodies with numerous functional modalities of oligonucleotides has been fruitful for a variety of applications with AOC including imaging, detection and targeted therapeutics. [1] [2] [3] Cell uptake/internalisation still represents the biggest hurdle towards successful ON ...