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Patients are about to be recruited for the first study to test a gene editing technique called CRISPR inside the body to try to cure a form of hereditary blindness.
People with the disease have normal eyes but do not have a gene that converts light into brain signals that allow vision.
The experimental treatment aims to provide children and adults with a healthy version of the gene that they lack, with the help of a tool to cut or "edit" the DNA at a specific place. . It is a punctual treatment that constantly modifies the native DNA of the person.
Two companies, Editas Medicine and Allergan, will test this system for up to 18 people in the United States, including Mbadachusetts Eye and Ear in Boston, starting this fall.
This genetic modification in individuals after birth is different from the controversial work done by a Chinese scientist last year – altering the embryo's DNA during conception so as to transmit the changes to future generations. The modifications of DNA in the adult that the new study aims to bring will not be inherited from any offspring.
Only one other company, Sangamo Therapeutics, has tried gene editing in the body to treat metabolic diseases using a tool called zinc fingers.
CRISPR appealed to scientists because it is a very simple way of editing genes, although it is so new that its risks are not fully known. But researchers think that it has great potential to cure or treat many diseases caused by genetic flaws that do not have good treatments now.
The blindness study is about people with a unique form of Leber conbad amaurosis. This is the most common cause of childhood hereditary blindness, which occurs in about 2 to 3 births per 100,000. People often only see bright light and fuzzy shapes and may eventually lose their sight.
The parents carry the defective gene and it can hide without being detected for generations. It suddenly appears when an unlucky combination gives two copies to a child.
A gene therapy called Luxturna is already sold for other forms of the disease and the treatment is similar: a modified virus is used to transport the replacement gene into retinal cells located at the back of the body. # 39; s eye. It is administered by injection during a short-term surgical procedure
The new study will test children aged 3 years and older and adults with a range of vision.
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Marilynn Marchione can be followed at http://twitter.com/MMarchioneAP .
The Health and Science Department of the Associated Press is receiving support from the Howard Hughes Medical Institute's Department of Science Education. The AP is solely responsible for all content.
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