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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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Size and Structure of Viral Genomes01:26

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Viral genomes exhibit remarkable diversity in size, structure, and composition, influencing their replication strategies and interactions with host cells. These genomes consist of either DNA or RNA and may be linear or circular. Additionally, they can be single-stranded or double-stranded, with each configuration affecting how the virus propagates within a host. RNA viruses, for instance, generally have smaller genomes than DNA viruses, a factor that contributes to their high mutation rates and...
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Viral Recombination00:57

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Cells are sometimes infected by more than one virus at once. When two viruses disassemble to expose their genomes for replication in the same cell, similar regions of their genomes can pair together and exchange sequences in a process called recombination. Alternatively, viruses with segmented genomes can swap segments in a process called reassortment.
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RNA Editing02:23

RNA Editing

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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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Viral Structure00:56

Viral Structure

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Viruses are extraordinarily diverse in shape and size, but they all have several structural features in common. All viruses have a core that contains a DNA- or RNA-based genome. The core is surrounded by a protective coat of proteins called the capsid. The capsid is composed of subunits called capsomeres. The capsid and genome-containing core are together known as the nucleocapsid.
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Genomics02:02

Genomics

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Genomics is the science of genomes: it is the study of all the genetic material of an organism. In humans, the genome consists of information carried in 23 pairs of chromosomes in the nucleus, as well as mitochondrial DNA. In genomics, both coding and non-coding DNA is sequenced and analyzed. Genomics allows a better understanding of all living things, their evolution, and their diversity. It has a myriad of uses: for example, to build phylogenetic trees, to improve productivity and...
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Related Experiment Video

Updated: Feb 4, 2026

Author Spotlight: Intrathecal Injection – An Efficient and Reliable Delivery Method to Test the Efficacy of Gene Editing in Neonatal Mouse Brains
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Author Spotlight: Intrathecal Injection – An Efficient and Reliable Delivery Method to Test the Efficacy of Gene Editing in Neonatal Mouse Brains

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Non-Viral Delivery To Enable Genome Editing.

Yuan Rui1, David R Wilson2, Jordan J Green3

  • 1Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.

Trends in Biotechnology
|October 4, 2018
PubMed
Summary

Genome editing tools like CRISPR offer revolutionary gene modification for treating diseases. Overcoming delivery challenges is key to translating these powerful biotechnologies for patient care.

Keywords:
CRISPR/CasTALENZFNgene editingnon-viral

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Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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Area of Science:

  • Molecular Biology
  • Biotechnology
  • Genetic Engineering

Background:

  • Genome-editing technologies, including zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR-associated protein systems, have transformed biological research.
  • These systems utilize DNA-binding proteins to induce site-specific double-strand breaks (DSBs) for precise gene modification.
  • The therapeutic potential of genome editing lies in correcting aberrant gene expression to cure genetic diseases.

Purpose of the Study:

  • To review the challenges associated with the delivery of genome-editing tools into the cell nucleus.
  • To highlight recent advancements in non-viral delivery methods for genome-editing technologies.
  • To discuss the potential of these innovations in advancing genome editing for clinical applications.

Main Methods:

  • Review of current literature on genome-editing technologies (ZFNs, TALENs, CRISPR-Cas).
  • Analysis of challenges in intracellular delivery, focusing on nuclear entry.
  • Examination of emerging non-viral delivery strategies and their efficacy.

Main Results:

  • Delivery into the cell nucleus remains a significant hurdle for therapeutic genome editing.
  • Non-viral delivery methods show promise in overcoming current limitations.
  • Innovations in delivery systems are crucial for the clinical translation of genome editing.

Conclusions:

  • Safe and efficient delivery is paramount for the therapeutic success of genome-editing technologies.
  • Advancements in non-viral delivery systems are critical for moving genome editing from research to patient care.
  • Further development in delivery strategies will accelerate the application of genome editing in treating genetic disorders.