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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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An Ex Vivo Choroid Sprouting Assay of Ocular Microvascular Angiogenesis
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CRISPR Technology for Ocular Angiogenesis.

Sook Hyun Chung1, Tzu-Ni Sin1, Taylor Ngo1

  • 1Department of Ophthalmology and Vision Science, University of California, Davis, Sacramento, CA, United States.

Frontiers in Genome Editing
|October 29, 2021
PubMed
Summary
This summary is machine-generated.

CRISPR gene editing offers a permanent solution for ocular angiogenesis, a leading cause of blindness. This review explores its potential to overcome limitations of current anti-VEGF treatments for complex eye diseases.

Keywords:
CRISPRVEGFangiogenesisanti-VEGFchoroidal neovascularizationgenome editingretinaretinal neovascularization

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Area of Science:

  • Ophthalmology and Gene Therapy
  • Genome Engineering and Molecular Biology

Background:

  • Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) systems are advanced genome engineering tools utilized for in vivo gene editing in ophthalmic conditions.
  • Current treatments for neovascular retinal diseases, including retinopathy of prematurity, proliferative diabetic retinopathy, and age-related macular degeneration, rely on frequent anti-vascular endothelial growth factor (anti-VEGF) injections.
  • These conventional therapies are often short-lived, costly, and burdensome for patients, highlighting the need for more sustainable solutions.

Purpose of the Study:

  • To review the evolution of gene therapy and advancements in adapting CRISPR platforms for suppressing retinal angiogenesis.
  • To discuss the potential of CRISPR technology as a permanent therapeutic strategy for complex, multifactorial ocular angiogenesis.
  • To compare CRISPR-based genome editing with conventional gene therapies for multifactorial versus monogenic retinal disorders.

Main Methods:

  • Review of existing literature on CRISPR applications in ophthalmology and gene therapy.
  • Discussion of various Cas9 orthologs and delivery strategies for in vivo genome editing.
  • Analysis of different genomic targets, including VEGF, VEGF receptor, and HIF-1α, for suppressing angiogenesis.

Main Results:

  • CRISPR technology presents a potential for permanent suppression of ocular angiogenesis by targeting intracellular signals and regulatory elements.
  • The technology allows for cell-specific delivery and multiplexing to disrupt multiple pro-angiogenic factors simultaneously.
  • Challenges include the permanent alteration of physiologic pathways, unpredictable editing efficacy, and potential off-target effects.

Conclusions:

  • CRISPR-based genome editing holds significant promise for developing permanent treatments for ocular angiogenesis, surpassing limitations of current anti-VEGF therapies.
  • Further research is needed to address safety concerns, optimize delivery, and understand the long-term implications of permanent pathway suppression.
  • Overcoming these barriers is crucial for the effective clinical adoption of CRISPR strategies in managing complex retinal vascular diseases.