Physicians need patients with clinically classic RP to consider clinical trials for gene therapy or gene therapy combined with other ophthalmic treatments. Patients with mild and asymptomatic RP, whose vision has not dropped or is improving, can often be assured of avoiding unnecessary research and treatment. Physicians should also consider clinical trials for patients who have some form of RP-associated dysmorphism.
Retinitis pigmentosa is a group of rare genetic diseases caused by mutations in genes that either lead to or cause the deterioration of the retina. \n
People with RP may have vision changes that can be described at 1 metre and 6 metres. There can be visual symptoms such as night blindness, loss of central acuity and tunnel vision. Symptoms are progressive and occasional in nature. There can be more subtle aspects to retinitis pigmentosa that manifest as difficulty with reading, driving, concentrating, and night sight problems.
RP is caused by mutations in more than 200 different genes. The genetic causes have many distinct types but are not well understood. Many genes are needed, both to specify the different kinds of RP and to explain the range of types and severity of the disease. Although a small number of patients can be traced back to a single person, most RP families are not related. This suggests genetic heterogeneity. There is also evidence that RP has a substantial environmental component, although the genetic and environmental causes are not fully understood. Even though the causes of RP are not well understood, it is possible to improve the lives of patients and protect their family members. This article focuses on the most common RP.
This novel gene therapy successfully rescued retinal degeneration caused by a well-defined mutation of rhodopsin gene and could reduce the development of retinal degeneration in a mouse model by 90%. The successful results of our therapy indicate the possible clinical usefulness of this treatment strategy for treating patients with ARMDs.
One study found at least 1.78 million individuals living with RP in the United States. The prevalence of RP is roughly 1 in 5000 in New England and about 1 in 50,000 in North America, while, in the rest of the country, it is about 1 in 125,000.
In a survey of adult and pediatric ophthalmologists, vitamin A-containing supplements are the most frequently used treatment. Immunoglobulin therapy is another frequent use. The use of laser treatment was very common.
Genetic:Aav5-rpgr 4e11 is typically used in combination with any other treatment. Gene correction using Lentiviral vectors seems to be of value in patients even when aav5 is not corrected. Retinal stem cells have been successfully used in patients with RP. The most success seems to have been observed in patients having a mutation in rhodopsin (typically rhodopsin p158h). Stem cell therapy was observed to work by creating a more efficient method of protein synthesis in the retina. With this new method of protein synthesis, the patient is then able to produce more protein than they were previously able to. Stem cell therapy and genetic therapy are two promising methods for improving retinal regeneration.
We have found that RP is frequently inherited as a dominant form. As with RDS, the family study is the best method for deciding the appropriate mode of inheritance.
This article summarizes the new findings in the field of retinitis pigmentosa and reviews the research in the area of ocular therapeutics. At this early stage of research, there is no treatment yet that totally prevents the development of RP. Researchers are working to find ways to help the patient’s retina, which would help preserve vision even at the initial stage, and is making huge progress. As the research progresses, we will be able to treat more and more patients. New types of therapies can be developed to treat retinitis pigmentosa. It is really nice to hope for the future and the future vision in patients with retinitis pigmentosa.
New drugs and therapies are constantly being researched. It is reported that some are already being tested on humans. Gene therapy is also in early tests. There is also a treatment that uses stem cells. These treatments are being used on patients to restore some sight. However, it can be difficult for patients to take the pills sometimes, and other patients may have difficulty taking pills forever to stay alive. There is a new treatment for patients with retinal degenerations because more and more patients with retinal dystrophies are diagnosed and it is difficult to heal them, because they have to have a long way to come back from blindness. This is because the eyeball may need to be replaced by surgery, eye implants, gene therapy, or laser.
Here we describe a novel, homologous AAV5 based gene therapy platform that allows gene transfer across a wide range of human cell types in vitro and in vivo. Although the current vector is relatively simple and can be used without refinement, additional changes to improve AAV5 transduction in vivo and in particular targeting of transgene expression towards photoreceptors in diseased models would be of major clinical benefit.