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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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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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CRISPR01:59

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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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CRISPR and crRNAs02:53

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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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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Homologous Recombination02:31

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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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Experimental RNAi02:15

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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: Jan 14, 2026

CRISPR Epigenome Editing in Human Cells using Plasmid DNA Transfection and mRNA Nucleofection Delivery
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A Chemically Induced CRISPR/dCas13FCPF Platform for Precise and Programmable RNA Regulation.

Sebastian Hasselbeck1,2, Jianhui Wang1,2, Zhaodai Bai3,4

  • 1Buchmann Institute for Molecular Life Sciences, Goethe University Frankfurt am Main, 60438 Frankfurt am Main, Germany.

Journal of Medicinal Chemistry
|October 22, 2025
PubMed
Summary
This summary is machine-generated.

Chem-CRISPR/dCas13FCPF precisely targets RNA for splicing modulation, using lower drug doses and reducing off-target effects. This novel chemical-genetic approach offers a controllable method for RNA modulation in therapeutics.

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Gene Digital Circuits Based on CRISPR-Cas Systems and Anti-CRISPR Proteins
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Gene Digital Circuits Based on CRISPR-Cas Systems and Anti-CRISPR Proteins

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Area of Science:

  • Molecular Biology
  • RNA Therapeutics
  • Gene Editing

Background:

  • Alternative splicing regulates protein diversity but its dysregulation is linked to diseases like cancer and neurodegeneration.
  • Current small-molecule splicing modulators lack specificity, causing off-target effects.
  • CRISPR/Cas13 systems offer transcript-level targeting but dCas13 fusion effectors can be large and disrupt RNA homeostasis.

Purpose of the Study:

  • To develop a precise and controllable RNA modulation system by combining CRISPR targeting with small-molecule action.
  • To create a modular platform, Chem-CRISPR/dCas13FCPF, for targeted splicing modulation with enhanced specificity and reduced dosage.

Main Methods:

  • Developed Chem-CRISPR/dCas13FCPF, a system covalently linking a small molecule to dCas13 via an FCPF π-clamp tag.
  • Utilized CRISPR RNA (crRNA) to guide the dRfxCas13dFCPF effector to the SMN2 exon 7 splice region.
  • Assessed splicing modulation by measuring exon inclusion and off-target effects on known sensitive transcripts.

Main Results:

  • Chem-CRISPR/dCas13FCPF induced SMN2 exon 7 inclusion at ligand doses approximately 500-fold lower than free risdiplam.
  • The system demonstrated no detectable off-target effects on other risdiplam-sensitive transcripts in the assays.
  • The platform showed generalizability to other transcripts through crRNA redesign.

Conclusions:

  • Chem-CRISPR/dCas13FCPF provides a proximity-induced, chemically controllable method for precise RNA modulation.
  • This approach couples CRISPR's targeting capability with dose-sparing chemical action for potential therapeutic applications.
  • The system represents a significant advancement in developing targeted RNA-based therapies with improved specificity and reduced side effects.