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The New Era of Therapeutic Strategies for the Management of Retinitis Pigmentosa: A Narrative Review of the Pathomolecular Mechanism for Gene Therapies
被引:1
|作者:
Nair, Praveena P.
[1
,2
]
Keskar, Manjiri P.
[3
]
Borghare, Pramod T.
[4
]
Dzoagbe, Hellen Y.
[5
]
Kumar, Tanish
[6
]
机构:
[1] Mandsaur Inst Ayurved Educ & Res, Otolaryngol, Bhunyakhedi, India
[2] Parul Univ, Parul Inst Ayurved, Otolaryngol, Limda, India
[3] Parul Univ, Parul Inst Ayurved, Otolaryngol, Limda, India
[4] Mahatma Gandhi Ayurved Coll Hosp & Res, Otolaryngol, Wardha, India
[5] Datta Meghe Inst Higher Educ & Res, Anat, Wardha, India
[6] Datta Meghe Inst Higher Educ & Res, Med, Wardha, India
关键词:
rhodopsin (rho) gene;
genetic mutations;
pathomolecular mechanisms;
genome editing;
stem cell;
transplantation;
photoreceptor degeneration gene therapy;
hereditary retinal degeneration;
retinitis pigmentosa;
DEGENERATION;
MUTATIONS;
DISEASE;
ABCA4;
D O I:
10.7759/cureus.66814
中图分类号:
R5 [内科学];
学科分类号:
1002 ;
100201 ;
摘要:
Retinitis pigmentosa, or RP, is a group of inherited retinal degenerations involving progressive loss of photoreceptor cells- rods and cones- ultimately causing severe vision loss and blindness. RP, although a very common ailment, continues to be an incurable disease with little to be done medically. However, with the breakthroughs in gene therapy and stem cell transplantation in recent years, a new door has been opened to the treatment of RP. This narrative review summarizes the pathomolecular mechanisms of RP, focusing on the genetic and molecular abnormalities that lead to the process of retinal degeneration. In this section, we talk about the current theories of how RP develops, gene mutations, oxidative stress, and inflammation. We also delve into new therapeutic approaches such as gene therapy, stem cell transplantation and genome surgery, which are designed to either replace or repair the damaged photoreceptors to restore vision and ultimately enhance the life of the RP patient. Another topic covered is the obstacles and research frontiers of these revolutionary treatments. This article is intended to give a complete overview of the molecular processes of RP and the promising treatment strategies that could change the way this devastating disease is treated.
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