U.S. researchers recently reported in the journal Science Translational Medicine that after treatment with genetic therapy, three patients with hereditary eye diseases had significantly improved their visual acuity and had no adverse reactions such as rejection. This brings hope for using gene therapy to treat other retinopathy.
The 3 patients had a rare eye disease--Lehbert Congenital Cataract. This disease usually occurs when the patient is young, and a gene of the patient's retinal cells mutates, causing severe vision loss and abnormal eye movements. By the age of 20 or 30, the patient may be completely blind. Researchers at the University of Pennsylvania and the Children's Hospital of Philadelphia used an adenovirus vector to implant the modified gene RPE65 into the eye of the patient. The virus would infect retinal diseased cells and "rewrite" the variant gene to a normal gene.
Out of caution, the researchers initially treated only one eye with poor vision in each patient. After several years of observation to confirm the safety and effectiveness, the researchers used the same method to perform another eye on the patient. treatment. After treatment, the patient can recognize the human face, which cannot be done before the treatment, and the patient's sensitivity to low light is significantly increased.
The 3 patients had a rare eye disease--Lehbert Congenital Cataract. This disease usually occurs when the patient is young, and a gene of the patient's retinal cells mutates, causing severe vision loss and abnormal eye movements. By the age of 20 or 30, the patient may be completely blind. Researchers at the University of Pennsylvania and the Children's Hospital of Philadelphia used an adenovirus vector to implant the modified gene RPE65 into the eye of the patient. The virus would infect retinal diseased cells and "rewrite" the variant gene to a normal gene.
Out of caution, the researchers initially treated only one eye with poor vision in each patient. After several years of observation to confirm the safety and effectiveness, the researchers used the same method to perform another eye on the patient. treatment. After treatment, the patient can recognize the human face, which cannot be done before the treatment, and the patient's sensitivity to low light is significantly increased.
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