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New form of therapy could avoid blindness in young people
The most common cause of blindness in young people is a disease called retinitis pigmentosa. Researchers now found that a new form of gene therapy in young people can help treat this disease. Thus, many cases of blindness may be avoided in the future.
Researchers at the internationally recognized University of Oxford found in an investigation that gene therapy can lead to successful treatment of a particular form (XLRP) of retinitis pigmentosa. The doctors published a press release on the results of their study.
A disease causes affected young people to gradually become blind. Researchers have developed a therapy that seems to enable successful treatment. (Image: Dan Race - fotolia) What is retinitis pigmentosa?
An inheritance or a spontaneously occurring mutation can cause a so-called retinal degeneration. In this disease, the photoreceptors are then damaged and destroyed. Retinitis pigmentosa is currently considered untreatable and causes a slow and irreversible loss of vision, the experts explain. On March 16, 2017, a 29-year-old British man was the first patient to begin a new form of gene therapy. The necessary surgery took place at the Oxford Eye Hospital.
Scientists insert a copy of a gene into the cells
Gene therapy uses a virus to insert the correct copy of a defective gene into the cells. The results indicate that this therapy appears to be effective in treating genetic causes of blindness, the researchers report.
Gene therapy is particularly challenging at RPGR
Unfortunately, the gene that affects retinitis pigmentosa (RPGR) is very unstable. This means that gene therapy is particularly demanding, explain the doctors. The unusual genetic code of the RPGR gene has made it very difficult for experts to work with him in the lab.
Genetic code from RPGR has been reprogrammed.
That's why the research team, under the direction of Professor Robert MacLaren of the University of Oxford, reprogrammed RPGR's genetic code. This makes it more stable without affecting its functions, explain the scientists. This change will allow the gene to be reliably delivered by a viral vector in retinal cells.
New sequence used is extremely effective in laboratory studies
"The impact of RPGR-related diseases on families with retinitis pigmentosa is devastating and we have spent many years developing this gene therapy," explains Professor MacLaren. Changing the genetic code is always done with great caution, but the new sequence used has proven very effective in laboratory studies, adds the author.
Gene is very unstable and prone to mutations
The genetic code for all life on Earth consists of four letters - G, T, A, and C. In RPGR, however, half the gene is just two letters - A and G, the researchers explain. This makes the gene very unstable and prone to mutations. These are a leading cause of blindness in patients with retinitis pigmentosa. RPGR is crucial for the photosensitive cells at the back of the eye, the physicians add.
Further research is needed
We are pleased to report the progress of this exciting gene therapy program to patients, say the experts. The current study will involve at least 24 patients to receive a single subretinal injection of RPGR gene therapy. If successful, this gene therapy has the potential to change the lives of many patients (and their families) around the world, the authors add. The primary goal of the study is to assess the safety and tolerability of this gene therapy over a 12-month period. (As)