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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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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.
RNA Performs Diverse...
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siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
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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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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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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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Updated: Oct 2, 2025

Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells
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Dual CRISPR-Interference Strategy for Targeting Synthetic Lethal Interactions Between Non-Coding RNAs in Cancer Cells

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Long Noncoding RNAs as Therapeutic Targets.

Jacob B Pierce1,2, Haoyang Zhou1,3, Viorel Simion1

  • 1Department of Medicine, Cardiovascular Division, Brigham and Women's Hospital, Harvard Medical School, Boston, MA, USA.

Advances in Experimental Medicine and Biology
|February 27, 2022
PubMed
Summary
This summary is machine-generated.

Long noncoding RNAs (lncRNAs) are key in cell function and disease. Advances in oligonucleotide therapies and delivery systems show promise for targeting lncRNAs to treat various diseases.

Keywords:
Cardiovascular diseaseDelivery platformsOligonucleotidesRNATherapeutics

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Area of Science:

  • Molecular Biology
  • Genetics
  • Therapeutics

Background:

  • Long noncoding RNAs (lncRNAs) regulate cellular functions and disease progression.
  • Their specific expression patterns make them attractive therapeutic targets.

Purpose of the Study:

  • To review recent advances in therapeutic strategies targeting lncRNAs.
  • To highlight successful pre-clinical applications and future challenges.

Main Methods:

  • Oligonucleotide chemistry modifications (e.g., 2'-fluoro, LNA, cEt, phosphorothioate bonds).
  • Novel delivery platforms (viral vectors, nanoparticles).
  • Oligonucleotide conjugation (e.g., peptide nucleic acids).

Main Results:

  • Therapeutic targeting of lncRNAs has shown efficacy in pre-clinical models for cardiovascular disease, sepsis, autoimmune disorders, and cancer.
  • Improved oligonucleotide chemistry and delivery platforms enhance therapeutic efficacy and pharmacokinetics.

Conclusions:

  • Significant progress has been made in lncRNA therapeutics, with promising pre-clinical results.
  • Overcoming remaining challenges in delivery and chemistry is crucial for clinical translation.