What is a good referense to review about ribozyme history and applications?5 answersA comprehensive reference for reviewing the history and applications of ribozymes can be found in various research papers. Ribozymes, catalytic RNA molecules, have shown promise in cancer therapeutics, genetic diseases, and infectious diseases caused by viral and bacterial pathogens. They have been utilized for gene therapies targeting genetic disorders, viral infections, and cancers, with trans-splicing ribozymes offering selective repair and reprogramming abilities in target RNA-expressing cells. Additionally, self-cleaving ribozymes have been engineered as biomolecular tools for diverse applications, including mRNA regulation and processing in various organisms. For a detailed overview of ribozyme history, characteristics, mechanisms of action, and current status as therapeutic agents, these papers provide valuable insights into the development and potential challenges faced by ribozymes in becoming effective molecular drugs.
What is the mechanism of action of hydrogels?5 answersHydrogels function through a crosslinked polymeric network that retains an optimal amount of water, providing biocompatibility, biodegradability, and hydrophilicity. They can absorb bodily fluids and mimic the extracellular matrix, making them versatile for various applications, including wound healing. Hydrogels can be chemically or physically crosslinked, allowing for the incorporation of therapeutic agents like drugs, natural extracts, and cells, which can be released in a controlled manner. Stimuli-responsive hydrogels, capable of changing properties in response to internal/external stimuli, enhance therapeutic effects, such as pro-neurogenesis, anti-inflammatory, anti-apoptosis, anti-oxidation, and pro-angiogenesis, particularly in traumatic brain injury treatment. Additionally, hydrogels can be tailored for passive or active drug delivery, depending on the application, route of administration, and type of drug.
Who discovered catalytic RNA (ribozymes)?5 answersCatalytic RNA, also known as ribozymes, was discovered by researchers in the field of molecular biology. The discovery of ribozymes introduced a new class of enzymes to biology, playing crucial roles in the RNA World hypothesis. Since the first RNA with catalytic activity was found in 1982, numerous ribozymes have been reported, showcasing their diverse functions in biological processes. Ribozymes catalyze various chemical reactions and are essential for the survival and evolution of biological entities, potentially playing a significant role in the primordial RNA world. The study of ribozymes provides insights into the foundational principles of biological catalysis and the intricate relationship between RNA structure and function.
What are aptazymes?4 answersAptazymes are synthetic molecules that consist of an aptamer domain and a catalytic active nucleic acid unit, such as a ribozyme or a DNAzyme. The aptamer domain serves as a molecular switch that can regulate the catalytic activity of the nucleic acid unit. This regulation is triggered by the binding of the aptamer to its target molecule, causing significant structural changes in the aptazyme. Aptazymes function similarly to allosteric enzymes, where the binding of ligands to allosteric sites alters the three-dimensional structure of the active site. Aptazymes have been used in various applications, including analytical assays, gene expression regulation, and as biosensors for the real-time detection of pathogens.
What are the advantages of RNA therapy?5 answersRNA therapy offers several advantages in the field of therapeutics. Firstly, RNA-based modalities such as mRNA, miRNA, antisense oligonucleotide (ASO), siRNA, and circular (circRNA) provide high flexibility in designing RNA and rapid production for clinical screening. Secondly, RNA drugs have the potential to target heterogeneous tumors with multiple sub-clonal cancer cell populations, making them suitable for precision medicine approaches. Additionally, RNA-based therapies allow for tissue-specific targeting, improving safety and reducing the concentration of target proteins selectively. They also offer more precise control of metabolic pathways compared to small molecule-based drugs and can reduce pill burden with less frequent administration schedules. Furthermore, RNA therapies have the advantage of being customizable and can be edited in various forms, such as secondary and tertiary structures, to enhance their effectiveness. Overall, RNA therapy holds promise for revolutionizing the treatment of various diseases, including cancer and lipid disorders.
What are the most promising therapeutic targets for RNA-based pharmacotherapy for tumors?3 answersRNA-based pharmacotherapy for tumors has shown promise in targeting specific genes and pathways involved in cancer. Some of the most promising therapeutic targets include cancer-associated genes, RNA splicing, and the epidermal growth factor receptor (EGFR). Aberrant expression of cancer-associated genes and RNA splicing has been implicated in the pathogenesis of various cancers, making them attractive targets for RNA therapeutics. Additionally, the dysregulation of EGFR is prevalent in glioblastoma multiforme (GBM), and targeting this gene has the potential to block the production of cancer-driving genes. By developing RNA therapies that interact with specific elements of the EGFR pre-mRNA transcript, it is possible to alter gene expression and potentially activate the expression of therapeutic isoforms. These targets offer opportunities for the development of RNA-based therapeutics that can effectively treat tumors.