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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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lncRNA - Long Non-coding RNAs02:39

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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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Types of RNA01:23

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 the regulation of 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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Types of RNA01:20

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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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MicroRNAs01:22

MicroRNAs

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MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
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Targeting noncoding RNAs in disease.

Brian D Adams, Christine Parsons, Lisa Walker

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    Regulatory RNAs, like microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), are key in chronic diseases. Targeting these noncoding RNAs with new therapies shows promise for treating various conditions.

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    Area of Science:

    • Biomedical Science
    • Molecular Biology
    • Genetics

    Background:

    • Regulatory RNAs, including microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), are increasingly recognized for their roles in chronic diseases.
    • Aberrant expression of miRNAs is implicated in cancer, cardiac dysfunction, metabolic disorders, and neurodegenerative diseases.

    Purpose of the Study:

    • To summarize the current landscape of targeting noncoding RNAs for therapeutic purposes.
    • To highlight the recent advancements and existing challenges in developing noncoding RNA-based therapies.

    Main Methods:

    • Review of recent scientific literature on noncoding RNA therapeutics.
    • Analysis of clinical trial data for antisense oligonucleotide-based therapies.
    • Discussion of RNA modification and delivery technologies.

    Main Results:

    • Over 100 antisense oligonucleotide therapies have entered clinical trials in the last 5 years, with a significant portion progressing to later phases.
    • FDA-approved therapies like fomivirsen and mipomersen demonstrate the clinical viability of targeting specific RNA molecules.
    • Advancements in RNA modifications and delivery systems, such as nanoparticles, are crucial for future therapeutic development.

    Conclusions:

    • Targeting noncoding RNAs represents a promising therapeutic strategy for a wide range of chronic diseases.
    • Continued innovation in RNA-based therapies is essential to overcome current challenges and expand treatment options.