Antisense oligonucleotides are small molecules that can be used to modulate gene expression by binding to specific RNA molecules. The correct spelling of this term is /æntisaɪns ɒlɪɡoʊˌnju:klɪətaɪdz/ using the International Phonetic Alphabet (IPA). "Antisense" is pronounced as /æntisaɪns/, while "oligonucleotides" is pronounced as /ɒlɪɡoʊˌnju:klɪətaɪdz/. The use of IPA helps to clarify the correct pronunciation of technical terms, which is important for effective communication within the scientific community.
Antisense oligonucleotides (ASOs) are short segments of synthetic nucleic acids, typically composed of 15 to 30 nucleotide bases, designed to selectively bind and modulate the expression of target genes. They are named "antisense" because they are complementary in sequence to specific messenger RNA (mRNA) molecules, which are responsible for translating the genetic information from DNA into functional proteins. ASOs exert their activity by binding to the complementary mRNA sequence, thereby hindering its normal function.
ASOs have emerged as a powerful tool in molecular biology and therapeutic research. By specifically targeting and binding to the mRNA molecules that code for disease-associated proteins, ASOs can effectively prevent the synthesis of these proteins within cells. This targeted approach enables researchers to investigate the essential role of specific genes and their corresponding proteins in various cellular processes and disease pathogenesis.
In the context of therapeutics, antisense oligonucleotides hold great promise as a class of drugs known as gene silencers or gene expression inhibitors. They can be designed to selectively block the expression of disease-causing genes, which may contribute to the development of conditions such as cancer, genetic disorders, and viral infections. Additionally, ASOs can modulate gene expression by promoting alternative splicing, leading to the production of different protein isoforms with altered functional properties.
ASOs are typically delivered into cells via various methods, such as direct injection, conjugation with cell-penetrating peptides, or encapsulation in targeted nanoparticles. Ongoing research and clinical trials are exploring the potential of antisense oligonucleotides as a novel therapeutic approach to treat a wide range of diseases.
The term "antisense oligonucleotides" is a combination of three components: "anti", "sense", and "oligonucleotides".
1. "Anti": It comes from the Greek word "anti" which means "opposite" or "against". In scientific terminology, "anti-" is commonly used to indicate an opposing action or effect.
2. "Sense": In molecular biology, the term "sense" refers to a coding sequence of DNA or RNA that can be translated into a functional protein. Conversely, the complementary strand to the sense sequence is called the "antisense" strand.
3. "Oligonucleotides": This term is formed by combining "oligo", meaning "few" or "small", and "nucleotides", which are the building blocks of DNA and RNA.