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Therapeutic Nucleic Acids


Nucleic acids are classified into two groups, deoxyribonucleic acid (DNAs) and ribonucleic acid (RNAs). These molecules are simple linear polymers that consist of four major subunits. They are always being the subject of considerable research and have now been moving in the field of alone therapeutic products. Therapy with nucleic acids either employs unmodified DNA/RNA or closely related chemical compounds. From both development and regulatory perspective, they fall to the extent between small molecules and biological agents. With their high specificity, functional diversity, and limited toxicity, therapeutic nucleic acids (TNAs) hold enormous promise for the treatment of several pathologies, infections, and even cancers. This realm coverage involves:

Nucleic acid-based therapeutic strategies. Figure 1. Nucleic acid-based therapeutic strategies. (Chakravarthy, 2017)

Development & Advancement

A series of critical discoveries in the last decades has made TNA possible. Firstly, a large body of work has clearly followed the decoding of the human genome that uncovered some molecular pathways that are important in the disease. Secondly, in addition to mRNA and tRNA, several types of RNAs with complex functions have been found, such as RNAi. Thirdly, the appreciation that RNAs perform as enzymes has resulted in the research and development of RNA analogs with useful, valuable, or unique properties. The fourth major advance has been improving the deliverable and bioavailability of nucleic acid-based drugs.

None of these would have been available without technological developments in DNA synthesis, including the de novo synthesis of increasingly longer DNA constructs and usage of DNA shuffling, bioprospecting, high-throughput screening, genetic engineering of viruses, and combinatorial chemistry. Advances in systems biology, synthetic biology, computational biology, bioinformatics, and nanotechnology have greatly promoted the progress in this area and settle a novel paradigm for the nucleic acids in therapy.

DNA Therapeutics

Human gene therapy takes advantage of new genetic material into the cells of an organism to offer a therapeutic benefit for the patient. Nucleic acids are one of the most significant sources not only for the understanding of the basic information of human life but also for the development of a promising candidate for therapeutics. One of the important advantages of DNA-based drugs over recently effective low molecular weight (MW) pharmaceuticals is their selective recognition of molecular targets and signal pathways, which imparts enormous specificity of action. DNA-based therapies contain several categories listed below and in gene therapy, gene transfer technology is commonly a DNA delivery system for TNAs.

RNA Therapeutics

There are two distinct classes of therapies that target nucleic acids, single-stranded ASOs and double-stranded RNAi molecules that operate through an interference pathway in the cells. The concept of RNA-targeting therapeutics, using siRNAs, miRNAs, ASOs, aptamers, and synthetic mRNAs, to control the expression of disease-related genes as a way to treat illness or disorder is an absorbing one. Such drugs have the ability to engage targets that are otherwise ‘undruggable’ by proteins and small molecules, thereby opening up completely new avenues for treating intractable diseases.

Most RNA therapeutics can be sorted into one of three broad categories, including those that target nucleic acids (either DNAs or RNAs), those that target proteins, as well as those that encode proteins. Composed methods that hybridize certain RNA-based machinery into a single package are also emerging. The fundamental approaches used in RNA therapies are in the following summary.

TNA Research & Development

The range of diseases that can potentially be solved by nucleic acids is wide and extends from infections to diabetes, cancers, etc. Key issues that need to be figured out for the successful translation of nucleic acids may be the choice between direct delivery and the option of a vehicle, their delivery to the site of action, mass production at low cost, ability to produce sustained long-term effects, more clearly defined pharmacokinetics (PK), toxicity & safety, and immunogenicity. Studies addressing these areas are needed to improve success in clinical trials. There is no doubt that an increased understanding of the biology of different nucleic acids will lead to hopeful therapies in the future.

Nucleic acids carry out myriad functions both within the cells and in the labs. Early analysis of their activity was rather narrow and restricted, but increasing studies have witnessed remarkable progress in developing nucleic acid-based therapeutics. Today, Creative Biolabs is always concentrating on the current status of nucleic acid therapies and keeping a watchful eye on the recent developments in TNA research.

If additional help is needed, please directly contact us and consult our technical supports for more details.

Reference

  1. Chakravarthy, M.; et al. (2017). Nucleic acid-based theranostics for tackling Alzheimer's disease. Theranostics. 7(16): 3933-3947.

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