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Related Concept Videos

RNA Interference01:23

RNA Interference

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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
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RNA Structure01:23

RNA Structure

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Overview
The basic structure of RNA consists of a five-carbon sugar and one of four nitrogenous bases. Although most RNA is single-stranded, it can form complex secondary and tertiary structures. Such structures play essential roles in the regulation of transcription and translation.
Different Types of RNA Have the Same Basic Structure
There are three main types of ribonucleic acid (RNA): messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). All three RNA types consist of a...
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Therapeutic Index01:13

Therapeutic Index

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The therapeutic index of a drug is a key parameter in pharmacology that quantifies the relative safety of a drug by calculating the ratio between the dose that causes toxicity in half the population (50%) to the dose that proves to be effective for half the population (50%). It provides a spectrum of doses for a particular drug ranging from effective to potentially toxic. To illustrate, consider an anticoagulant agent like warfarin. It possesses a narrow window within its therapeutic index to...
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RNA Stability01:53

RNA Stability

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Intact DNA strands can be found in fossils, while scientists sometimes struggle to keep RNA intact under laboratory conditions. The structural variations between RNA and DNA underlie the differences in their stability and longevity. Because DNA is double-stranded, it is inherently more stable. The single-stranded structure of RNA is less stable but also more flexible and can form weak internal bonds. Additionally, most RNAs in the cell are relatively short, while DNA can be up to 250 million...
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RNA Splicing01:32

RNA Splicing

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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Related Experiment Video

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TRUE Gene Silencing: Screening of a Heptamer-type Small Guide RNA Library for Potential Cancer Therapeutic Agents
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Tapping the RNA world for therapeutics.

Judy Lieberman1

  • 1Program in Cellular and Molecular Medicine, Boston Children's Hospital and Department of Pediatrics, Harvard Medical School, Boston, MA, USA. judy.lieberman@childrens.harvard.edu.

Nature Structural & Molecular Biology
|April 18, 2018
PubMed
Summary

Recent advances in RNA biology have revolutionized therapeutic development. RNA-based drugs offer a promising new frontier, targeting nucleic acids to expand treatment options beyond traditional protein targets.

Area of Science:

  • Molecular Biology
  • Pharmacology
  • Drug Discovery

Background:

  • Recent breakthroughs in RNA biology have uncovered novel RNA classes, modifications, and transcriptional activities.
  • These discoveries have catalyzed a paradigm shift in developing RNA-based therapeutic strategies.

Purpose of the Study:

  • To review the current landscape of RNA-based drug design.
  • To identify and discuss challenges hindering wider therapeutic applications.
  • To propose potential solutions for overcoming these barriers.

Main Methods:

  • Literature review of recent advancements in RNA biology.
  • Analysis of current RNA-based therapeutic modalities.
  • Discussion of challenges and future directions in RNA drug development.

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Main Results:

  • Identification of new RNA classes and functions.
  • Emergence of novel RNA modification types.
  • Recognition of widespread extragenic transcription and alternative splicing.

Conclusions:

  • RNA-based therapeutics represent a significant advancement, expanding the scope of druggable targets.
  • Overcoming current barriers is crucial for realizing the full potential of RNA-based drugs.
  • Targeting nucleic acids offers a powerful alternative to conventional small molecule and biologic drugs.