Related Experiment Video
Updated: Jul 3, 2025
08:46
Implementation of In Vitro Drug Resistance Assays: Maximizing the Potential for Uncovering Clinically Relevant Resistance Mechanisms
Published on: December 9, 2015
10.6K
RNA therapeutics history and future perspectives
Hong-Quan Duong1, Minh-Cong Hoang2, Thi-Hue Nguyen3
1Hanoi University of Public Health, Hanoi, Vietnam.
Progress in Molecular Biology and Translational Science
|February 15, 2024
Summary
RNA therapeutics, including messenger RNA vaccines, offer new treatment options for diseases conventional drugs cannot address. These advancements provide novel avenues for preventing and treating various human conditions.
Area of Science:
- Biotechnology
- Molecular Biology
- Pharmacology
Background:
- Ribonucleic acid (RNA) therapeutics have emerged as a significant advancement in medicine.
- Their development has been accelerated by successes like messenger RNA (mRNA) vaccines during the COVID-19 pandemic.
- RNA-based drugs offer alternatives for diseases unresponsive to traditional treatments.
Purpose of the Study:
- To provide a comprehensive overview of RNA therapeutics.
- To detail the history, mechanisms, and applications of various RNA-based drug classes.
- To highlight advancements in RNA delivery methods for targeted treatment.
Main Methods:
- Review of historical RNA biology discoveries and their therapeutic implications.
- Analysis of approved RNA-based drugs, their mechanisms of action, and therapeutic targets.
- Discussion of current and emerging RNA delivery systems for various diseases.
Main Results:
- RNA therapeutics encompass five main classes: antisense oligonucleotides, small interfering RNA, microRNA, aptamers, and mRNA.
- These therapeutics are effective against a range of diseases, including neurological, cardiovascular, genetic, rare diseases, and cancers.
- Significant progress has been made in developing effective delivery methods for RNA therapeutics.
Conclusions:
- RNA therapeutics represent a groundbreaking approach to treating diverse human diseases.
- The field continues to evolve, offering new hope for conditions with limited treatment options.
- Continued research into RNA biology and delivery systems promises further therapeutic breakthroughs.
More Related Videos
Related Concept Videos
02:57Targeted Cancer Therapies
7.6K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
There are several types of targeted therapies against...
7.6K
01:31Transducer Mechanism: Nuclear Receptors
1.3K
Nuclear receptors, or NRs, are unique transcription factors that regulate gene transcription and affect the cellular pathways involved in reproduction, development, or metabolism. Their ability to be stimulated by small lipophilic ligands and control vital cellular processes makes them ideal drug targets. Nearly 10-15% of currently prescribed drugs target these receptors.
About 48 different soluble family members of nuclear receptors are identified that can be divided into two main classes:
About 48 different soluble family members of nuclear receptors are identified that can be divided into two main classes:
1.3K
01:27Transducer Mechanism: Enzyme-Linked Receptors
2.5K
Enzyme-linked receptors are cell-surface receptors acting as an enzyme or associating with an enzyme intracellularly. They make excellent drug targets. Drugs can bind to the extracellular ligand-binding domain or directly affect their enzymatic domain and alter their activity.
Major types that are helpful drug targets include:
Major types that are helpful drug targets include:
2.5K
01:26Drug Administration and Therapy Phases: Overview
465
Drugs, the chemical agents used in diagnosing, treating, or preventing diseases, undergo a four-phase process of development: pharmaceutic, pharmacokinetics, pharmacodynamics, and therapeutic.
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
The pharmaceutical phase focuses on leveraging the physicochemical properties of the drug to design and manufacture an effective product. Variants include orally administered tablets or capsules, topical creams or ointments, and parenteral-delivery solutions or emulsions.
The pharmacokinetic phase...
465
00:59Gene Therapy
25.4K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.4K
01:28Structure-Activity Relationships and Drug Design
720
Drug design is a dynamic field that involves discovering and developing new medications based on specific biological targets. This process heavily relies on structure-activity relationships (SAR) and quantitative structure-activity relationships (QSAR) to guide the design and optimization of efficient drugs.
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence...
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence...
720