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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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RNA Pull-down Procedure to Identify RNA Targets of a Long Non-coding RNA
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RNA Pull-down Procedure to Identify RNA Targets of a Long Non-coding RNA

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RNA-Targeted Therapeutics.

Stanley T Crooke1, Joseph L Witztum2, C Frank Bennett1

  • 1Ionis Pharmaceuticals, Inc., 2855 Gazelle Court, Carlsbad, CA 92010, USA.

Cell Metabolism
|April 5, 2018
PubMed
Summary
This summary is machine-generated.

RNA-targeted therapies, using modified oligonucleotides and Watson-Crick base pairing, have overcome significant hurdles. Approved for conditions like Spinal Muscular Atrophy (SMA), this drug discovery platform efficiently targets previously undruggable diseases.

Keywords:
GalNAcRNAianti-miRsantisenseoligonucleotidesiRNA

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • RNA-targeted therapies utilize chemically modified oligonucleotides and Watson-Crick base pairing to interact with cellular RNAs.
  • Significant challenges in this field have been successfully surmounted, paving the way for therapeutic advancements.

Purpose of the Study:

  • To summarize the progress and current state of RNA-targeted therapies.
  • To outline the future challenges and opportunities within this drug discovery platform.

Main Methods:

  • The study reviews the development and application of chemically modified oligonucleotides as therapeutic agents.
  • It highlights the use of Watson-Crick base pairing as a target-binding motif for RNA interaction.

Main Results:

  • Four RNA-targeted therapies are currently approved for conditions such as Spinal Muscular Atrophy (SMA) and for reducing low-density lipoprotein cholesterol (LDL-C).
  • These therapies are administered via various routes, including subcutaneous, intravitreal, and intrathecal delivery, demonstrating technological efficiency.
  • The platform enables targeting of previously
  • undruggable
  • targets, with three additional agents in registration and more in clinical development.

Conclusions:

  • RNA-targeted therapies represent a mature and efficient drug discovery platform with a growing pipeline.
  • Continued progress in understanding molecular mechanisms is enhancing clinical performance and expanding therapeutic possibilities.