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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/Cas9 Genome Editing01:28

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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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Homologous Recombination02:31

Homologous Recombination

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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: Dec 25, 2025

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
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CRISPR Interference-Potential Application in Retinal Disease.

Caroline F Peddle1, Lewis E Fry1,2, Michelle E McClements1

  • 1Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford OX3 9DU, UK.

International Journal of Molecular Sciences
|April 2, 2020
PubMed
Summary
This summary is machine-generated.

CRISPR interference offers a precise and reversible method to silence disease-causing genes in inherited retinal diseases. This gene-editing tool shows promise for developing safer therapies by avoiding permanent genomic alterations.

Keywords:
CRISPR interferenceCRISPR/Cas9CRISPRiKRABRNAidCas9gene therapyknock-downretinal diseasetranscriptional repression

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Area of Science:

  • Genetics
  • Ophthalmology
  • Molecular Biology

Background:

  • Dominantly inherited retinal diseases necessitate silencing of pathogenic alleles.
  • Current gene silencing methods like RNA interference have off-target effects, and CRISPR-mediated gene disruption causes permanent genomic changes.

Purpose of the Study:

  • To review the potential of CRISPR interference (CRISPRi) as a therapeutic strategy for autosomal dominant inherited retinal diseases.
  • To discuss the advantages of CRISPRi over existing gene silencing techniques and address challenges for clinical translation.

Main Methods:

  • CRISPR interference utilizes a catalytically inactive 'dead' Cas9 protein guided by RNA to block gene transcription without DNA alteration.
  • Review of pre-clinical studies demonstrating CRISPRi's efficacy in gene silencing both in vivo and ex vivo.

Main Results:

  • CRISPRi is highly specific and potentially reversible, offering a safer therapeutic profile.
  • Pre-clinical studies show CRISPRi's versatility for gene silencing in various contexts, including iPSC modification for transplantation.

Conclusions:

  • CRISPR interference presents a promising, targeted approach for treating autosomal dominant inherited retinal diseases.
  • Further in vivo studies are needed to overcome challenges and advance CRISPRi towards clinical application in ophthalmology.