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Related Concept Videos

CRISPR01:59

CRISPR

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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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Related Experiment Video

Updated: Sep 12, 2025

Investigation of the Transcriptional Role of a RUNX1 Intronic Silencer by CRISPR/Cas9 Ribonucleoprotein in Acute Myeloid Leukemia Cells
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Systematic screening for functional exon-skipping isoforms using the CRISPR-RfxCas13d system.

Qiang Sun1, Xuejie Ma2, Qianqian Ning3

  • 1Cancer Institute, Xuzhou Medical University, Xuzhou 221004, Jiangsu, China; Center of Clinical Oncology, the Affiliated Hospital of Xuzhou Medical University, Xuzhou 221004, Jiangsu, China; Jiangsu Center for the Collaboration and Innovation of Cancer Biotherapy, Cancer Institute, Xuzhou Medical University, Xuzhou 221004, Jiangsu, China; Center for RNA Medicine, the Fourth Affiliated Hospital of School of Medicine, International School of Medicine, International Institutes of Medicine, Zhejiang University, Yiwu 320000, Zhejiang, China.

Cell Systems
|August 5, 2025
PubMed
Summary
This summary is machine-generated.

This study introduces a new CRISPR tool to silence specific cancer-driving gene variants. Researchers identified exon skipping events, like HMGN3 Δ6, crucial for colorectal cancer growth and progression.

Keywords:
CRISPR-RfxCas13d screeningHMGN3Δ6colorectal cancerexon skipping

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Area of Science:

  • Molecular Biology
  • Genomics
  • Cancer Research

Background:

  • Exon skipping (ES) is a common splicing alteration in cancer, but its functional impact is poorly understood.
  • Understanding ES-derived isoforms is crucial for cancer biology and therapeutic development.

Purpose of the Study:

  • To develop a transcript-specific gene silencing platform for studying ES events.
  • To identify functional ES events driving colorectal cancer (CRC) progression.

Main Methods:

  • Developed a CRISPR-RfxCas13d system for targeting exon-exon junctions.
  • Created a transcriptome-wide guide RNA library for 3,744 human ES events.
  • Performed loss-of-function screens in CRC cells (in vitro and in vivo).

Main Results:

  • Identified multiple ES events critical for CRC cell growth.
  • Discovered HMGN3 Δ6, an ES isoform, significantly enhances tumor proliferation.
  • Validated the oncogenic role of HMGN3 Δ6 in CRC progression.

Conclusions:

  • CRISPR-RfxCas13d is effective for isoform-specific functional genomics.
  • ES-derived transcripts represent a significant, understudied layer of tumor biology.
  • ES events, like HMGN3 Δ6, are potential therapeutic targets in cancer.