23.12.2016

American Researchers Discover Novel Gene Editing Technology For Curing Blindness And Much More

Genetic science is advancing at an astonishing pace nowadays. Many scientists actually believe that they have figured out how to manipulate our DNA so everyone can live healthier and longer lives.

The new therapy geneticists are so excited about is in the field of "gene editing." This therapy is concerned with changing how genes in vital organs like the brain, liver, and heart work. Oftentimes, the root of genetic illnesses is found in these vital organs. Until today, scientists have not been able to manipulate the genes in these organs due to the fact that the cells located there don't divide.

This breakthrough in gene editing therapy is coming out of Salk Institute for Biological Studies in La Jolla, California. Researchers at Salk say they are the first in the world to effectively cut through DNA samples in organs without dividing cells and replace them with healthy genetic codes.

Although Salk researchers have only used their technology on mice thus far, they are confident they can transfer these findings on to humans at some point in the future. Salk scientists call their particular brand of gene therapy "HITI," or more accurately homology-independent targeted integration, and they have used this therapy to cure many mice of blindness.

The mice in this experiment were all born with a form of genetic blindness called retinitis pigmentosa. Researchers effectively created a DNA-repair pathway and then put in brand new DNA into the eyes. Everyone involved in this study was shocked to see these mice naturally regain their sense of sight almost overnight.

Professors working on this HITI therapy are very excited with what this therapy might hold for the future of medicine. Professor Juan Carlos Izpisua Belmonte, who was a head researcher on HITI, told reporters that this kind of technology is a first in the gene editing world. He actually believes curing blindness is only the tip of the iceberg in terms of what this therapy could do for mankind.

It isn't only American researchers, however, who are interested in gene editing. There have been many reports of Chinese researchers working on DNA modifying techniques. Their most famous form of DNA editing is called CRISPR. These Chinese scientists have shown that CRISPR can re-grow human skin and heal damage done to the human gut.

When asked how CRISPR works, the Chinese researchers say that they use the cells' natural copying mechanisms to their advantage. These researchers just add the DNA they have copied and manufactured into certain areas of a person's genome.

Salk researchers knew about the CRISPR experiments before their own tests. Professors at Salk maintain that their experiments with HITI are at least ten times more powerful and effective than CRISPR.

Researchers at Salk have made such a bold statement on their HITI technology because they believe they are the first people in the world to successfully help regions of the body where cells don't divide. This has huge implications for how doctors treat areas of the body such as the eyes, heart, pancreas, and even the brain.

More tests need to be done to ensure the HITI will be just as effective on humans as it currently is on mice. Researchers are now working on improving the delivery efficiency of the HITI.


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