Introduction
CRISPR is a new technology that has scientists very excited. It allows them to make very precise changes to DNA, the genetic material that determines how all living things grow and function. And now, scientists are using CRISPR to cure blindness in mice!
What is CRISPR?
CRISPR stands for “Clustered Regularly Interspaced Short Palindromic Repeats.” It’s a mouthful, but it basically means that scientists can use a special protein to cut DNA at very specific points. This makes it possible to add, remove, or change small pieces of genetic material. It’s kind of like using a pair of scissors to cut out a single word from a book, and then replacing it with a different word.
How does CRISPR cure blindness in mice?
One of the most exciting things about CRISPR is that it has the potential to cure genetic diseases. That’s because many diseases are caused by a mistake or mutation in a person’s DNA.
Retinitis pigmentosa (RP) is a genetic disease that causes blindness by damaging the light-sensing cells in the retina. Gene editing tools like CRISPR-Cas have shown promise in correcting the mutations responsible for RP, but they have limitations in targeting specific regions in the genome. A new study has developed a genome-editing tool called PESpRY, which combines a few different techniques. The PESpRY system was used to correct the mutation in the gene in the neural retinas of a mouse model of RP, leading to improved vision. This study provides a new approach to targeted gene editing in retinitis pigmentosa and other inherited retinal disorders.
Right: Cut-away view of an eye with Retinitis Pigmentosa
Scientists are using CRISPR to cut out the mutated part of the gene, and replace it with a healthy copy. This restores the gene’s function, and allows the mouse to see again!
What does this mean for humans?
While this research is still in its early stages, it’s very promising. If scientists can use CRISPR to cure blindness in mice, there’s a good chance they could do the same thing in humans. Of course, there are still many questions that need to be answered before that can happen. Scientists need to make sure that the treatment is safe, effective, and doesn’t cause any unintended consequences. But if all goes well, CRISPR could be the key to curing many genetic diseases in the future.
CRISPR is a powerful new technology that allows scientists to make precise changes to DNA. By using CRISPR to cure blindness in mice, scientists have shown that it has the potential to cure genetic diseases in humans as well. While there is still a lot of research to be done, this is an exciting step forward in the field of genetic medicine.
Check out the full study in the Journal of Experimental Medicine (https://rupress.org/jem/article/220/5/e20220776/213949/Vision-rescue-via-unconstrained-in-vivo-prime)