Therapeutic RNA targeting involves the use of RNA molecules or RNA-targeting drugs to modulate gene expression or correct genetic disorders, offering a precise approach to treat diseases at their molecular root. This strategy leverages the natural roles of RNA in gene regulation and protein synthesis, enabling the development of highly specific therapies for a wide range of conditions, including cancers, viral infections, and genetic diseases.

Therapeutic RNA Targeting

RNA molecules

RNA-targeting drugs

Gene expression is the process by which information from a gene is used to synthesize a functional gene product, typically proteins, which ultimately determine cellular function and phenotype. This process is tightly regulated at multiple levels, including transcription, RNA processing, translation, and post-translational modifications, to ensure proper cellular function and response to environmental cues.

Gene Expression

Genetic disorders are diseases caused by abnormalities in an individual's DNA, which can be inherited or occur spontaneously. They can result from mutations in a single gene, multiple genes, or be due to chromosomal abnormalities and can vary greatly in severity and symptoms.

Genetic Disorders

Diseases at their molecular root

Natural roles of RNA

Gene regulation is the process by which cells control the expression and timing of genes to ensure proper function and adaptation to environmental changes. This complex system involves multiple mechanisms that can activate or suppress gene activity at various stages, from transcription to post-translational modifications.

Gene Regulation

Protein synthesis is the cellular process through which genetic information is translated into functional proteins, essential for cellular structure and function. It involves two main stages: transcription, where DNA is converted into mRNA, and translation, where mRNA is decoded by ribosomes to assemble amino acids into polypeptide chains.

Protein Synthesis

Highly specific therapies

Cancers are a group of diseases characterized by the uncontrolled growth and spread of abnormal cells, which can form tumors, invade adjacent parts of the body, and metastasize to other organs. Understanding the molecular mechanisms behind cancer development and progression is crucial for developing effective treatments and preventive strategies.

Cancers

Viral infections occur when viruses invade the body's cells and replicate, often causing disease by damaging or killing host cells and triggering immune responses. They can spread through various means, such as direct contact, airborne transmission, or vectors, and are typically managed by vaccines, antiviral drugs, and supportive care.

Viral Infections

Genetic diseases are disorders caused by abnormalities in an individual's DNA, which may be inherited or result from new mutations. These diseases can affect various bodily functions and systems, with severity ranging from mild to life-threatening conditions.

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