JAN 14, 2016 02:45 PM PST

Gene Editing To Solve a Common Cause of Blindness

WRITTEN BY: Kara Marker
A disease that causes blindness in both rats and humans could be prevented or reversed with a new genetic editing technology known as CRISPR/Cas9.
 
Inherited retinitis pigmentosa, a group of eye diseases causing “progressive vision loss” as cells of the retina gradually deteriorate, affects about 1 in 4,000 people in the United States (NIH). In a recent study of rat models of the disease, scientists from Cedars-Sinai Medical Center capitalized on CRISPR/Cas9 abilities to disable the genetic mutation responsible for blindness in this disease. Their study was recently published in the journal Molecular Therapy.
 
"This is the first time CRISPR/Cas9 gene editing has been used to prevent vision loss in a living animal,” said Clive Svendsen, PhD, a co-author of the study.
 
CRISPR/Cas9 was implemented based on a protective action of bacteria in the face of invading viruses. Bacteria replicate viral genes upon invasion and copy them into an RNA sequence. Then, the RNA binds to a protein called Cas9 and guides it to the gene in the virus so Cas9 can disable the gene.
 

The research team for this study repurposed the CRISPR/Cas9 technology to target the rhodopsin gene, which carries the dominant S334ter mutation that causes photoreceptor loss in the retina. After disabling the mutated gene in the rat models of disease, the researchers compared their new visual abilities to control rats without the disease by testing their optomoter reflex, an evaluation of visual acuity (Vision Research).
 
The study concluded that with a CRISPR/Cas9 treatment to disable the trouble-causing mutation, the rats were able to see better.
 
Although rare overall, inherited retinitis pigmentosa is a very common cause of blindness in the United States, and there is no current cure. With potential applications for human cases of inherited retinitis pigmentosa, this new finding creates a promising future for the treatment of this disease.

Watch the following video to see what it looks like to have vision impaired by inherited retinitis pigmentosa.
 

Source: Cedars-Sinai Medical Center
 
About the Author
  • I am a scientific journalist and enthusiast, especially in the realm of biomedicine. I am passionate about conveying the truth in scientific phenomena and subsequently improving health and public awareness. Sometimes scientific research needs a translator to effectively communicate the scientific jargon present in significant findings. I plan to be that translating communicator, and I hope to decrease the spread of misrepresented scientific phenomena! Check out my science blog: ScienceKara.com.
You May Also Like
NOV 20, 2018
Immunology
NOV 20, 2018
Mutations Mutations Which Ones Do We Want?
A team at UCSF makes use of new SLICE tool to generate mutations that reveal specific genetic functions....
NOV 24, 2018
Genetics & Genomics
NOV 24, 2018
Alzheimer's Researchers Detect Genetic Recombination in the Brain
Our genomic sequence is thought to remain the same throughout our lives. But new research has found evidence to the contrary....
DEC 28, 2018
Videos
DEC 28, 2018
What is Sanger Sequencing?
You may have heard of Sanger sequencing, a common tool used in the genetics laboratory to decode the sequence of a strand of DNA....
JAN 16, 2019
Genetics & Genomics
JAN 16, 2019
Gene Therapy for a Leading Cause of Infant Death is Closer to Market
New treatments for some diseases can have extremely high price tags. One example is Zolgensma, which might cost $5 million per patient....
JAN 21, 2019
Genetics & Genomics
JAN 21, 2019
Genetic Connections to Osteoarthritis and Potential Treatments
Scientists are learning more about how small genetic changes can lead to an increased risk of disease....
JAN 22, 2019
Genetics & Genomics
JAN 22, 2019
Towards a Better Understanding of Genetic Modifications in Plants
Scientists have taken a close look at what happens on the molecular level when DNA is inserted into the genome of a plant....
Loading Comments...