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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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Updated: Jun 1, 2025

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
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Recent progress in CRISPR/Cas9 system for eye disorders.

D A Ayush Gowda1, Girish Birappa1, Sripriya Rajkumar1

  • 1Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, South Korea.

Progress in Molecular Biology and Translational Science
|January 17, 2025
PubMed
Summary
This summary is machine-generated.

The CRISPR/Cas9 gene editing system shows promise for treating ocular disorders by correcting genetic mutations. This technology is advancing the development of new therapies for eye diseases.

Keywords:
BlindnessCRISPR/Cas9Clinical trialsEye diseaseGene editingNucleasesPathologyRetinal degenerationSingle guide RNATherapeuticsVision impairment

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Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Ocular disorders present diverse symptoms due to genetic and environmental factors.
  • The eye's unique structure supports advanced gene editing therapies.
  • Genome editing offers potential treatments for various diseases, including eye conditions.

Purpose of the Study:

  • To explore the application of CRISPR/Cas9 technology in ophthalmology.
  • To discuss the generation of ocular disease models using CRISPR/Cas9.
  • To review current and future therapeutic uses of CRISPR/Cas9 for eye pathologies.

Main Methods:

  • Utilizing the CRISPR/Cas9 system for gene editing in ocular research.
  • Generating ocular disease models in mammalian cell lines and in vivo.
  • Investigating the correction of mutations in patient-derived cells.

Main Results:

  • CRISPR/Cas9 enables gene modification in mammalian models of eye development and disease.
  • Successful correction of harmful mutations in patient-derived ocular cells.
  • Development of various ocular disease models for further study.

Conclusions:

  • CRISPR/Cas9 technology is a powerful tool for studying and potentially treating ocular disorders.
  • Further research is needed to overcome challenges and fully realize therapeutic potential.
  • This gene editing system holds significant promise for the future of ophthalmology.