N-Acetylcysteine (NAC) for Retinitis pigmentosa

被引:0
|
作者
Pfau, Kristina [1 ,2 ]
Callizo, Josep [1 ]
Rossouw, Petra [1 ]
Gabrani, Chrysoula [1 ]
Holz, Frank [2 ]
Issa, Peter Charbel [3 ]
Kellner, Ulrich [4 ]
Strauss, Rupert [5 ]
Kuehlewein, Laura [6 ]
Stingl, Katarina [6 ]
Feltgen, Nicolas [1 ]
Pfau, Maximilian [1 ,2 ,7 ]
机构
[1] Univ Spital Basel, Augenklin, Mittlere Str 91, CH-4056 Basel, Switzerland
[2] Univ Klinikum Bonn, Augenklin, Bonn, Germany
[3] Tech Univ Munich, Univ Klinikum, Klin & Poliklin Augenheilkunde, Munich, Germany
[4] AugenZentrum Siegburg, RetinaScience, Siegburg, Germany
[5] Med Univ Graz, Univ Augen Klin, Graz, Austria
[6] Univ Tubingen, Univ Augenklin, Dept Augenheilkunde, Tubingen, Germany
[7] Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland
关键词
Retina; Retinitis pigmentosa; retinal degeneration; antioxidants; inherited retinal diseases; retina; retinitis pigmentosa; CONE CELL-DEATH; OXIDATIVE STRESS; DAMAGE; GENE;
D O I
10.1055/a-2525-4419
中图分类号
R77 [眼科学];
学科分类号
100212 ;
摘要
Retinitis pigmentosa (RP) is a genetically mediated degenerative retinal disease that leads to progressive photoreceptor degeneration and, ultimately, blindness. Oxidative stress plays a central role in the pathogenesis of RP. This study examines the effect of N-acetylcysteine (NAC), an antioxidant and glutathione precursor, on the disease progression in RP patients. NAC can reduce oxidative stress in the retina and has shown potential neuroprotective effects in preclinical models. Initial clinical studies indicate improvements in visual acuity and macular function with NAC therapy, though no structural changes have yet been demonstrated. The ongoing multicentre Phase III trial "NAC Attack" aims to evaluate the long-term efficacy and safety of NAC in RP.
引用
收藏
页码:199 / 204
页数:6
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