Retinitis Pigmentosa: From Pathomolecular Mechanisms to Therapeutic Strategies

被引:6
|
作者
Vingolo, Enzo Maria [1 ]
Mascolo, Simona [1 ]
Micciche, Filippo [1 ]
Manco, Gregorio [1 ]
机构
[1] Univ Roma La Sapienza, Polo Pontino Osped A Fiorini, Sense Organs Dept, UOSD Ophtalmol, I-4019 Terracina, Italy
来源
MEDICINA-LITHUANIA | 2024年 / 60卷 / 01期
关键词
retinitis pigmentosa; inherited retinal disease; genotype; therapy; ERG; vitamin A; HBO; cells transplantation; AAV; HYPERBARIC-OXYGEN THERAPY; VISUAL-FIELD PROGRESSION; STEM-CELL THERAPY; DOCOSAHEXAENOIC ACID; CLINICAL-TRIAL; GENE-THERAPY; VITAMIN-A; RETINAL DEGENERATION; SUPPLEMENTATION; INJECTION;
D O I
10.3390/medicina60010189
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Retinitis pigmentosa is an inherited disease, in which mutations in different types of genes lead to the death of photoreceptors and the loss of visual function. Although retinitis pigmentosa is the most common type of inherited retinal dystrophy, a clear line of therapy has not yet been defined. In this review, we will focus on the therapeutic aspect and attempt to define the advantages and disadvantages of the protocols of different therapies. The role of some therapies, such as antioxidant agents or gene therapy, has been established for years now. Many clinical trials on different genes and mutations causing RP have been conducted, and the approval of voretigene nepavorec by the FDA has been an important step forward. Nonetheless, even if gene therapy is the most promising type of treatment for these patients, other innovative strategies, such as stem cell transplantation or hyperbaric oxygen therapy, have been shown to be safe and improve visual quality during clinical trials. The treatment of this disease remains a challenge, to which we hope to find a solution as soon as possible.
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页数:11
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