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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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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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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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RNA Editing02:23

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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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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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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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Updated: Nov 16, 2025

Functional Assessment of BRCA1 variants using CRISPR-Mediated Base Editors
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Development and Characterization of a Modular CRISPR and RNA Aptamer Mediated Base Editing System.

Juan Carlos Collantes1, Victor M Tan1, Huiting Xu1

  • 1Department of Pharmacology, Robert Wood Johnson Medical School, Piscataway, New Jersey, USA.

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A new base editing (BE) system, Pin-point, precisely modifies DNA without double-strand breaks (DSB). This offers a safer and more efficient alternative for genetic engineering and therapeutic development.

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Area of Science:

  • Molecular Biology
  • Gene Editing Technologies
  • Biotechnology

Background:

  • Conventional CRISPR gene editing uses DNA double-strand breaks (DSB), which can be genotoxic and inefficient in somatic cells.
  • Base editing (BE) systems offer an alternative by modifying target bases without inducing DSBs or relying on homology-directed repair (HDR).

Purpose of the Study:

  • To introduce a novel base editing system, Pin-point, for precise genome modification.
  • To evaluate the efficiency and precision of the Pin-point system in human cells.

Main Methods:

  • Development of the Pin-point system, which utilizes an RNA aptamer within the guide RNA (gRNA) to recruit a DNA base-modifying enzyme.
  • Assessment of base pair modification efficiency and on-target indel formation in the human genome.

Main Results:

  • The Pin-point system demonstrated efficient and precise modification of base pairs in the human genome.
  • Low on-target indel formation was observed with the Pin-point system.

Conclusions:

  • The Pin-point base editing system provides a precise and efficient method for genome editing without DSBs.
  • This technology holds potential for correcting pathogenic mutations, introducing stop codons, and advancing genetic research and therapeutic development.