Restoring Vision with the use of Landmark gene-editing technique

The Associated Press reports that a new study is about to be conducted on patients who have an inherited form of blindness. The study will test the gene-editing technique called CRISPR made famous by a Chinese scientist last year.

People who are born blind have a disease caused a lack of a gene that converts light into signals to the brain that enables normal vision.

A statement in NEWSER, an e-editorial, says that “the blindness study is for people with one form of Leber congenital amaurosis and it is the most common cause of inherited childhood blindness that occurs in about 2 to 3 of every 100,000 births. People with this disease often see only bright light and blurry shapes and eventually can lose all sight.”

About 18 children and adults will be signed up for the study and each will be supplied with a healthy version of the gene they lack. The gene-editing technique, CRSPR, is a tool that will cut or ‘edit’ DNA in a very specific location. This will permanently alter the person’s normal DNA and will be a onetime treatment.

The two companies who will do the testing using the CRSPR method will select people from around the United States. Editas Medicine and Allergan are the companies but Massachusetts Eye and Ear in Boston will also be involved.

Editas Medicine is based in Cambridge, MA and its specialty is in developing therapies based on CRISPR–Cas9  gene-editing technology.  Allergan is a pharmaceutical company based in Dublin, Ireland.

There is only one other US company called Sangamo Therapeutics, based in Brisbane, California, which has tried gene-editing inside the human body in order to treat metabolic diseases using a tool called zinc fingers. This gene-editing technique in people after birth is different from the very controversial work of He Jiankui, who is a Chinese biophysics researcher and who was an associate professor in the Department of Biology of the Southern University of Science and Technology located inShenzhen, China.

Jiankui said he used a technology known as CRISPR to edit (alter) the genes of human embryos that led to live births, something that had never been done before. This altering of genes atconception would pass the changes to future generations he said. Chinese investigators later said they confirmed his claim.

The DNA changes in people in this new study to correct blindness will not be inherited by their offsprings.

Many scientists believe that the CRISPR technique of gene-editing has great potential to cure many diseases caused by gene flaws that currently have no good treatments.