RNAs are multifunctional biomolecules that can be broadly defined as coding RNAs and non-coding RNAs (ncRNAs). ncRNAs are of various types, including tRNAs, lncRNAs, miRNAs, small interfering RNAs (siRNAs), saRNAs, circRNAs, and exosomal RNAs, among other types. Different RNA types have different applications in medicine. mRNAs can be used as therapeutics, diagnostic biomarkers, or therapeutic targets. In cells, mRNAs can be translated to produce therapeutic proteins to replace defective or missing proteins. mRNAs can also serve as therapeutic targets for antisense oligonucleotides (ASOs), siRNAs, miRNAs, aptamers, and inhibitory tRNAs.
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Simply put, DNA is a long sequence of four bases coded in an irregular order of A, T, G, and C. It is the carrier of genetic information in most organisms.The information coded in DNA can form genetic instructions that are used to guide the development of organisms and the operation of life functions. DNA bases in living organisms form a long sequence that almost never exists as a single strand, but instead applies the principle of base complementarity by forming base pairs, either A to T or T to A, G to C or C to G, to build up another sequence of DNA bases as a pair of closely related double strands that are intertwined with each other to form a double helix structure.
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DNA (deoxyribonucleic acid) and RNA (ribonucleic acid) are the two key nucleic acids in the cell, responsible for the storage and transmission of genetic information. DNA has A double helix structure, consisting of two long strands of deoxyribose and four bases (adenine A, thymine T, cytosine C and guanine G), and its main function is to store and protect genetic information. RNA, on the other hand, is A single-stranded structure containing ribosomes and four bases (adenine A, uracil U, cytosine C, and guanine G) that copies genetic information from DNA during transcription and then converts this information into proteins during translation. There are many types of RNA, including messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA), each carrying out different functions such as information transfer, amino acid transport, and protein synthesis. In general, DNA is the repository of genetic information, while RNA plays a direct role in protein synthesis.
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DNA (deoxyribonucleic acid) and RNA (ribonucleic acid) are two different nucleic acid molecules with distinct differences in structure and function.
DNA and RNA are the two basic molecules necessary for the storage and function of genetic information. Although they are evolutionarily related and function similarly in cellular processes, their differences in structure and characteristics allow them both to make specific contributions that will impact the field of molecular biology and hinder the development of therapies.
RNA-based therapies are an emerging and important new model for treating a wide range of diseases because they can target mechanisms related to gene expression and regulation. These therapies advanced rapidly before a more nuanced understanding of RNA biology and improvements in molecular techniques.
Small interfering RNAs (siRNAs) are double-stranded RNA molecules that trigger gene silencing through RNA interference processes and are important tools in functional genomics. Inside the cell, the sirna is loaded into the RNA-induced silencing complex (RISC) and guided to the target mrna by base pair matching. This means that this strategy allows for specific and effective gene silencing.
ASOs, short single-stranded nucleotides, are used to selectively bind to target mRNA molecules. This leads either to RNase H1-mediated degradation of target RNA or to changes in splicing patterns that affect gene expression. Some ASOs have been approved for the treatment of Duchenne muscular dystrophy and spinal muscular atrophy. So this is a beautiful demonstration of ASOs treating genetic diseases by targeting open sequences in RNA.
Aptamers, short RNA sequences that specifically bind to target proteins or small molecules. Due to its high specificity and affinity, scFvs are considered a suitable therapeutic or diagnostic tool. There are also RNa-based drugs, such as pegaptanib, an aptamer that inhibits vascular endothelial growth factor (VEGF) for the treatment of age-related macular degeneration.
mRNA vaccine is a new method of vaccination in which antigens are introduced through viral vectors formed by mRNA. Once inside the cell, the mRNA is translated into a target antigen, which stimulates an immune response.
RNA therapy is an innovative new class of drugs that target RNA molecules involved in disease pathways to treat a variety of diseases. Taken together, these observations suggest that ASOs, siRNAs, RNA aptamers, and mRNA vaccines could have a significant clinical impact. Although challenges in delivery, stability, and site-directed specificity still need to be addressed, advances in RNA technology offer opportunities for therapeutic applications by improving existing systems.
DNA-based therapy is a new, innovative gene therapy approach for treating a wide range of diseases. These therapies target diseases that, once a genetic basis is discovered, are labeled "incurable" because small-molecule drugs don't work and because people can't treat genetic mutations with drugs. Given the nature of DNA and its central role in directly regulating gene expression (through transcription) and gene repair (mutation reversal), it was obvious to explore this possibility.
These circular DNA molecules, called plasmids, act as vectors to transfer functional genes into target cells. These plasmids can carry transgenes, which are introduced into cells to produce therapeutic proteins or correct genetic defects.
In these systems, plasmid DNA encoding the antigen is introduced into the host cell, expressed through cellular mechanisms and elicited protein-specific immune responses. This has been studied in vaccines for infectious diseases and even cancer. The rapid development of DNA vaccines against SARS-CoV-2 demonstrates the potential of this strategy in addressing global health challenges.
DNA-based approaches, including gene therapy and DNA vaccines, continue to evolve, offering promising solutions for a range of medical conditions.