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CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration
Erin R Burnight1, Joseph C Giacalone1, Jessica A Cooke1
1Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, United States.
Progress in Retinal and Eye Research
|March 27, 2018
Summary
Gene correction offers hope for inherited retinal diseases. CRISPR-Cas9 gene editing and stem cell therapies are advancing treatments for blindness caused by genetic mutations.
Area of Science:
- Ophthalmology
- Genetics
- Regenerative Medicine
Background:
- Inherited retinal degenerative diseases are a leading cause of irreversible blindness worldwide.
- Single gene defects are responsible for most retinal dystrophies.
- Current gene augmentation therapies face challenges with large gene sizes, vector limitations, and potential retinal toxicity.
Purpose of the Study:
- To review current developments in CRISPR-Cas9 gene editing for inherited retinal diseases.
- To explore the application of gene editing and stem cell technologies in treating these conditions.
Main Methods:
- In vivo studies in animal models.
- In vitro studies using patient-derived cells.
- CRISPR-Cas9 gene editing.
- Induced pluripotent stem cell technology.
Main Results:
- CRISPR-Cas9 gene editing shows promise for correcting disease-causing mutations.
- Stem cell-based therapies offer potential for cell replacement in advanced disease stages.
- These technologies provide powerful tools for studying and developing treatments for retinal dystrophies.
Conclusions:
- CRISPR-Cas9 gene editing and stem cell technologies represent a significant advancement in treating inherited retinal degenerative diseases.
- These innovative approaches hold promise for restoring vision in patients with genetic blindness.
- Further research and clinical translation are crucial for realizing the full potential of these therapies.
Keywords:
CRISPR-Cas9Genome engineeringInduced pluripotent stem cellsRetinal degenerationTransplantationMore Related Videos
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