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Naked oligonucleotides are inherently prone to degradation and exhibit poor drug-like properties. The chemical modification is now one of the most promising ways to make oligonucleotide-based drugs deliver, maintain, and perform.
It is oligonucleotide therapeutics that have changed modern medicine, providing specialized treatments for previously incurable conditions. They are drugs - artificial RNA or hybrid RNA-DNA molecules that control RNA activity or protein levels.
Human genes are units of inheritance composed of DNA sequences that code for proteins or regulatory molecules that are necessary for the body to function. Humans have a genome with around 3 billion base pairs of DNA and around 20,000 or 25,000 genes.
Oligonucleotides are short synthetic nucleic acid polymers that generally have between 10 and 200 nucleotides. They can be single- or double-stranded, based on DNA or RNA, linked together by phosphodiester bonds.
Oligonucleotide therapy is a new precision medicine strategy in which short nucleotides are used to precisely block genes. These synthetic oligonucleotides are less than 20 nucleotides long and they can control gene expression by linking to Watson-Crick bases in DNA, mRNA, or pre-mRNA.
The 3'-protected oligomer is an oligonucleotide characterized by the presence of a protecting group on the 3'-hydroxyl group. The protecting group prevents non-specific reactions of the 3'-hydroxyl group in solid phase synthesis or subsequent modifications, improving synthetic efficiency and purity.
The 5'-DMTr-3'-OH oligomer is an oligonucleotide characterized by the presence of a 4,4'-dimethoxytrityl (DMTr) protecting group on the 5'-hydroxyl group and the presence of a free 3'-hydroxyl group.
Oligonucleotide synthesis is a sophisticated chemical technique used to construct short-stranded DNA or RNA molecules for a wide range of applications in biomedical research, molecular diagnostics and therapeutic drug development.
3'-phosphoramidite oligomers are essential intermediates in nucleic acid synthesis, providing unrivaled precision and efficiency in the assembly of DNA and RNA sequences. Their unique chemical design, especially the use of 3'-cyanoethyl (3'-CE) groups as the protecting part, greatly facilitates the automated synthesis of oligonucleotides.
Oligonucleotide synthesis is a critical process in molecular biology and pharmaceutical research, relying heavily on the use of 3'-protected monomers to achieve high precision, efficiency, and purity in the final products.
Linkers can broadly be categorized into non-cleavable and cleavable types, which are is crucial in determining the stability of ligand-oligonucleotide conjugates in plasma, as well as their biophysical properties, pharmacokinetic behavior, and therapeutic efficiency.
Alfa Chemistry is committed to collecting and updating information on the current state of the market and clinical development of oligonucleotide therapeutics, as this information plays a vital role in guiding ongoing research and development efforts.
Despite their great therapeutic potential, oligonucleotide therapeutics face several limitations or challenges. To develop oligonucleotide therapeutics, potency, specificity, and efficacious delivery issues must be addressed.
mRNA capping is a necessary post-transcriptional modification in eukaryotic cells that is involved in RNA stability, translation efficiency, and immune evasion.
CpG adjuvants are a class of immunostimulatory agents widely used in vaccine development and immunotherapy. They work primarily by stimulating Toll-like receptor 9 (TLR9), triggering involuntary immune responses, and increasing vaccine immunogenicity.
How nucleotides and nucleosides differ is the heart of biochemistry, molecular biology, and pharmacology. Firms such as Alfa Chemistry are critical in making these biomolecules available for studies and therapy.
Nusinersen (Spinraza) constitutes a breakthrough treatment option for spinal muscular atrophy (SMA), which is an inherited neurodegenerative disease that leads to motor neuron degeneration.
Recent years have seen oligonucleotides (oligos) emerge as important therapeutic agents for treating numerous ocular diseases that previously lacked effective treatment options.
Nucleosides represent fundamental biomolecules that consist of a nitrogenous base (purine or pyrimidine) linked to a five-carbon sugar (ribose or deoxyribose) via a β-glycosidic bond.
Protected nucleosides function as chemically altered nucleosides where specific functional groups like hydroxyl or amino groups receive temporary protection through protecting groups because they serve as important intermediates during nucleic acid synthesis.
As a specialist in oligonucleotide therapeutics, Alfa Chemistry offers consumers a wealth of raw materials and a full range of technologies and services.
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