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Genetic Lingo

Genetic Lingo

In-vitro Mutagenesis

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

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Related Experiment Video

Updated: Aug 6, 2025

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Enhanced Genome Editing with Cas9 Ribonucleoprotein in Diverse Cells and Organisms

Published on: May 25, 2018

Eye on genome editing.

Samuel W Du¹, Krzysztof Palczewski^1,2

¹Departments of Physiology & Biophysics and Ophthalmology, University of California, Irvine , Irvine, CA, USA.

The Journal of Experimental Medicine

|March 17, 2023

Summary

This summary is machine-generated.

CRISPR gene editing successfully restored vision in a mouse model of retinitis pigmentosa. This breakthrough utilized prime editing to correct genetic mutations causing inherited blindness.

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Last Updated: Aug 6, 2025

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CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.

CRISPR/Cas9 Editing of the C. elegans rbm-3.2 Gene using the dpy-10 Co-CRISPR Screening Marker and Assembled Ribonucleoprotein Complexes.

Published on: December 11, 2020

Area of Science:

Ophthalmology
Genetics
Molecular Biology

Background:

Inherited retinal diseases, such as retinitis pigmentosa, cause progressive vision loss and remain largely untreatable.
Genetic mutations are the primary cause of many inherited vision disorders.
CRISPR/Cas9 genome editing offers a potential therapeutic strategy for correcting causative mutations.

Purpose of the Study:

To investigate the efficacy of prime editing for correcting mutations causing retinitis pigmentosa.
To restore visual function in a preclinical model of inherited retinal disease.

Main Methods:

Utilized a prime editor system to target and correct the specific mutation in the rd10 mouse model of retinitis pigmentosa.
Assessed the restoration of visual function following gene editing treatment.

Main Results:

Successful correction of the genetic mutation responsible for retinitis pigmentosa in the targeted mouse model.
Significant restoration of visual functions was observed in the treated mice.

Conclusions:

Prime editing is a viable and effective tool for correcting genetic defects underlying inherited vision loss.
This study demonstrates the potential of CRISPR-based therapies for treating retinitis pigmentosa and similar genetic eye conditions.