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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.
The CRISPR-Cas system stores a copy of foreign DNA in the host genome and uses it to identify the foreign DNA upon reinfection. CRISPR-Cas has three different...
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Updated: May 29, 2025

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From bench to bedside: Developing CRISPR/Cas-based therapy for ocular diseases.

Qing Zhao1, Linxin Wei1, Youxin Chen1

  • 1Department of Ophthalmology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing 100730, China; Key Laboratory of Ocular Fundus Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College Hospital, Beijing 100730, China; Beijing Key Laboratory of Fundus Diseases Intelligent Diagnosis & Drug/Device Development and Translation, Beijing 100730, China.

Pharmacological Research
|January 31, 2025
PubMed
Summary
This summary is machine-generated.

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein (Cas) gene editing shows promise for treating vision-threatening diseases. Ongoing clinical trials and future research aim to overcome challenges for safe and effective ocular therapies.

Keywords:
CRISPR/CasGene deliveryGene therapyGenome editingOcular diseases

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Area of Science:

  • Ophthalmology
  • Genetics
  • Biotechnology

Background:

  • Vision-threatening disorders require novel therapeutic strategies.
  • CRISPR/Cas gene editing offers potential for ocular disease treatment.
  • CRISPR/Cas facilitates disease modeling and pathogenesis research.

Purpose of the Study:

  • To review CRISPR/Cas applications in ocular disease models and therapies.
  • To summarize CRISPR/Cas delivery methods and strategies.
  • To discuss current clinical trials and future directions for CRISPR/Cas in ophthalmology.

Main Methods:

  • Review of preclinical and clinical studies on CRISPR/Cas for ocular diseases.
  • Analysis of CRISPR/Cas-based disease models (animal, organoid, cell lines).
  • Examination of therapeutic targets including retinitis pigmentosa, glaucoma, and corneal diseases.

Main Results:

  • CRISPR/Cas has been used to develop disease models and explore therapeutic applications.
  • Preclinical studies show potential for restoring cellular function and alleviating disease phenotypes.
  • Active clinical trials target conditions like glaucoma, inherited retinal degenerations, and age-related macular degeneration.

Conclusions:

  • CRISPR/Cas technology holds significant promise for treating a range of ocular conditions.
  • Challenges such as off-target effects, immunogenicity, and regulatory hurdles need addressing.
  • Future research should focus on enhancing specificity, efficacy, and delivery for successful clinical translation.