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Related Concept Videos

lncRNA - Long Non-coding RNAs02:39

lncRNA - Long Non-coding RNAs

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In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA...
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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.
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Drug Discovery: Overview01:26

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Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
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Types of RNA01:20

Types of RNA

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Three main types of RNA are involved in protein synthesis: messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). These RNAs perform diverse functions and can be broadly classified as protein-coding or non-coding RNA. Non-coding RNAs play important roles in regulating gene expression in response to developmental and environmental changes. Non-coding RNAs in prokaryotes can be manipulated to develop more effective antibacterial drugs for human or animal use.
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Targets for Drug Action: Overview01:26

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Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
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Experimental RNAi02:15

Experimental RNAi

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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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Long non-coding RNA-targeting therapeutics: discovery and development update.

Olga Khorkova1, Jack Stahl1, Aswathy Joji1

  • 1Center for Therapeutic Innovation and Department of Psychiatry and Behavioral Sciences, University of Miami, Miami, FL, USA.

Expert Opinion on Drug Discovery
|July 19, 2023
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Summary
This summary is machine-generated.

Long non-coding RNAs (lncRNAs) are key regulators of cellular functions and emerging therapeutic targets. Nucleic acid-based therapies targeting lncRNAs show promise for treating genetic diseases and cancers.

Keywords:
Long non-coding RNAlncRNAnucleic acid-based therapeuticsoligonucleotidesregulation of expressionsiRNA

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Area of Science:

  • Molecular Biology
  • Genetics
  • Drug Development

Background:

  • Long non-coding RNAs (lncRNAs) are crucial regulators of cellular processes including gene expression and chromosomal interactions.
  • Their role in disease pathogenesis makes them attractive targets for novel therapeutic strategies.

Purpose of the Study:

  • To review recent advancements in targeting long non-coding RNAs (lncRNAs) for therapeutic applications.
  • To highlight the clinical potential of lncRNA-based therapeutics for unmet medical needs.

Main Methods:

  • Literature review of recent studies on lncRNA therapeutics.
  • Analysis of clinical applications and development trends in nucleic acid-based therapies.

Main Results:

  • lncRNAs offer a broad druggable target range with high specificity.
  • Nucleic acid-based therapeutics targeting lncRNAs show potential for genetic diseases, cancers, and protein deficiencies.
  • Advancements in delivery methods are overcoming administration challenges.

Conclusions:

  • lncRNA-targeting drugs are poised to become significant in future drug development, particularly in personalized medicine.
  • The clinical safety demonstrated by mRNA vaccines validates nucleic acid therapeutic technologies.