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A first study in patients suggests that a new genetic treatment for complete color blindness, developed by research groups in Tübingen and Munich, is safe.
Preliminary evidence of its effectiveness has also been obtained; people born with complete color blindness cannot distinguish colors.
His eyesight is blurred and his eyes are very sensitive to bright light.
This is due to a defect in the cone cells: the light receptors in the retina that are responsible for daylight and color vision.
Around 3,000 people in Germany are affected by complete color blindness, achromatopsia.
So far, there is no treatment for the underlying cause; in about a third of all achromatopsia patients, the defect is in the CNGA3 gene.
A team from the Ophthalmology Institute of the Tübingen University Hospital and the LMU Pharmacy and Ophthalmology Clinics have developed a therapy that, in principle, should remedy this genetic defect.
The normal version of the CNGA3 gene is introduced directly into the patient’s retina using a harmless virus.
After a few weeks, the cells of the retina can express this functional version of the CNGA3 gene and can produce the intact form of the corresponding protein, which should restore the function of the defective cones.
In this case, the healthy gene is carried by an adeno-associated virus developed by Professors Stylianos Michalakis and Martin Biel at LMU. The first clinical study of this approach in patients has just been completed at the Tübingen University Clinic.
The results appear in the journal JAMA Ophthalmology.
In the study, nine achromatopsia patients between the ages of 24 and 59 were injected with the virus with the CNGA3 gene intact in …
