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Related Concept Videos

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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MicroRNAs01:22

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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...
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Transfer RNA Synthesis02:36

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One of the unique features of tRNA is the presence of modified bases. In some tRNAs, modified bases account for nearly 20% of the total bases in the molecule. Altogether, these unusual bases protect the tRNA from enzymatic degradation by RNases.
Each of these chemical modifications is carried by a specific enzyme, post-transcription. All of these enzymes have unique base and site-specificity. Methylation, the most common chemical modification, is carried by at least nine different enzymes, with...
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Regulated mRNA Transport02:22

Regulated mRNA Transport

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Regulated mRNA Transport02:22

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In eukaryotes, transcription and translation are compartmentalized; an mRNA is first synthesized in the nucleus and then selectively transported to the cytoplasm for protein synthesis. Before transport, a pre-mRNA undergoes several steps of post-transcriptional modifications including splicing, 5' capping, and the addition of a poly-adenine tail. Various proteins bind to the pre-mRNA during these modifications. The mRNA transport takes place with the help of multiple proteins playing...
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Transcriptomic Analysis of Human Retinal Surgical Specimens Using jouRNAl
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mRNA transcript therapy.

Drew Weissman1

  • 1Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

Expert Review of Vaccines
|November 1, 2014
PubMed
Summary
This summary is machine-generated.

Messenger RNA (mRNA) therapeutics are revolutionizing medicine, overcoming previous challenges in immune stimulation and potency. This technology enables new applications from cancer vaccines to gene editing, with promising future prospects.

Keywords:
PKRmRNAmodified nucleosidepseudouridinepurificationtherapeutic proteinvaccine

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Therapeutic Technology

Background:

  • Messenger RNA (mRNA) is fundamental to life, with origins tracing back to an RNA world.
  • The therapeutic potential of mRNA was first described in 1992.
  • Recent advancements have addressed key challenges in mRNA delivery, namely immune stimulation and potency.

Purpose of the Study:

  • To review the current state of mRNA drug technologies.
  • To explore potential applications of mRNA therapeutics.
  • To discuss the challenges and future prospects in mRNA development and drug discovery.

Main Methods:

  • Literature review of mRNA therapeutic technologies.
  • Analysis of current clinical trial data for mRNA-based therapies.
  • Exploration of emerging applications and future research directions.

Main Results:

  • Significant progress has been made in overcoming immune stimulation and potency issues with mRNA.
  • mRNA technology is enabling diverse therapeutic strategies, including cancer immunotherapies.
  • Novel applications such as in vivo protein replacement, stem cell generation, and genome engineering are emerging.

Conclusions:

  • mRNA drug technology has matured, offering a versatile platform for various medical applications.
  • Overcoming delivery and stability challenges has unlocked the therapeutic potential of mRNA.
  • Continued research and development promise further breakthroughs in mRNA-based drug discovery and treatment.