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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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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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RNA Interference01:23

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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.
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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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Related Experiment Video

Updated: Jul 16, 2025

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms
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Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

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Precision RNA base editing with engineered and endogenous effectors.

Laura S Pfeiffer1, Thorsten Stafforst2,3

  • 1Interfaculty Institute of Biochemistry, University of Tübingen, Tübingen, Germany.

Nature Biotechnology
|September 21, 2023
PubMed
Summary
This summary is machine-generated.

RNA base editing precisely alters genetic information in RNA molecules for therapeutic applications. This review covers strategies, focusing on adenosine deaminases acting on RNA (ADAR) enzymes, for improved efficiency and in vivo delivery.

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Area of Science:

  • Molecular Biology
  • Biotechnology
  • Genetics

Background:

  • RNA base editing rewrites genetic information within RNA molecules.
  • Enzymes in human cells catalyze nucleobase conversions, enabling RNA recoding.
  • Unlike DNA editing, RNA editing is reversible and doseable, offering therapeutic potential.

Purpose of the Study:

  • To review current site-directed RNA base-editing strategies.
  • To highlight advancements in editing efficiency, precision, and delivery.
  • To discuss harnessing endogenous adenosine deaminases acting on RNA (ADAR) enzymes.

Main Methods:

  • Summarizing current site-directed RNA base-editing strategies.
  • Reviewing engineered editing effectors and endogenous ADAR enzyme strategies.
  • Analyzing achievements in improving editing efficiency, precision, and in vivo delivery.

Main Results:

  • Current RNA base editing focuses on adenosine and cytidine deamination.
  • Recent progress has enhanced editing efficiency, precision, and codon-targeting scope.
  • In vivo delivery into disease-relevant tissues is a key focus.

Conclusions:

  • RNA base editing offers reversible and doseable therapeutic applications.
  • The field anticipates the first RNA base-editing drug for genetic diseases soon.
  • Future challenges include optimizing RNA base editing for safe and doseable modulation of biological processes.