CRISPR Holy Grail of Gene Editing to Cure Blindness

dna crispr

A team of American scientists has discovered a revolutionary way to cure incurable diseases through gene alteration, which could potentially extend human lifespan.

Diseased cells in the liver, eyes, brain, and heart tend not to split, which has previously made access to find a cure impossible. However, researchers at the Salk Institute for Biological Studies in San Diego, California, believe that they have just found the ‘holy grail of gene editing’ and are now able to cut through DNA with precision.

What has been cured so far?

Chinese scientists have used a technique known as CRISPR, a DNA-modifying technique to divide cells in the skin or intestine, which is what inspired the new method that scientists have named HITI.

By finding a way to divide diseased cells in the eyes of blind laboratory mice the HITI method was successful at restoring a significant percentage of their vision, which was previously thought to be impossible. Rats and mice born with retinitis pigmentosa, a genetic disease that causes blindness, were used to prove that vision can be restored using this new method.

Professor Carlos Izpisua Belmonte, the lead researcher of the study, says: “This is nothing compared to the changes that this technique could demonstrate in the decades to come. We are very excited by the technology that we have discovered because it’s something that could not have been done before!”

How does it work?

“For the first time, we can enter into cells that do not divide and modify the DNA at will,” explained Professor Belmonte.

By using the normal copying mechanisms of the body’s cells, DNA can be inserted and added into the exact locations within each genome, which has proven to be a method ten times more efficient at incorporating new DNA into groups of dividing cells.

This is the first time in history that scientists have been able to access a precise DNA location and insert a new gene into it, especially in mature cells that no longer have dividing capabilities. Diseased cells of the eye, heart, brain, and pancreas could be restored to health by using this method, which is very exciting for the medical and scientific industries.

What’s next?

While the revolutionary procedure offers abundant possibilities for future disease treatment, the team’s next steps will be to improve the efficiency of the HITI construct delivery.

Their biggest challenge remains getting enough cells to incorporate the new DNA, which is a challenge across the board of genome-editing methods. The good news is that HITI technology can adapt to any type of gene-editing technique, something which the CRISPR method cannot do.

“We now have a technology that allows us to modify the DNA of non-dividing cells to fix broken genes in the brain, heart, and liver. It allows us, for the first time, to be able to dream of curing diseases that we couldn’t before, which is very exciting,” said Professor Belmonte.

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