Marks Erica, Anugu Adith, Bisiani Joseph, Pentyala Srinivas
Department of Anesthesiology, Renaissance School of Medicine at Stony Brook University, Stony Brook, NY, USA.
Department of Anesthesiology, Renaissance School of Medicine at Stony Brook University, 100 Nicolls Road, Stony Brook, NY 11794, USA.
Ther Adv Ophthalmol. 2025 May 23;17:25158414251320592. doi: 10.1177/25158414251320592. eCollection 2025 Jan-Dec.
Stargardt disease or Juvenile Macular Degeneration is a rare genetic disorder caused by a mutation in the ABCA4 gene that results in degeneration of the macula and loss of central vision. The mutation in the ABCA4 gene causes a build-up of lipofuscin, which is a substance that is left over from the breakdown and absorption of damaged blood cells. This build-up of lipofuscin causes the death of photoreceptor cells and the subsequent degeneration of the macula. Due to the nature of Stargardt's disease, there are currently no cures or treatment options. However, in recent years, there has been an increase in research and exploration of utilizing stem cell therapy as a potential cure and treatment for Stargardt disease. Growing research in the field of ophthalmology has found that the use of stem cells can potentially replace the loss of photoreceptor cells, slow the progression of the degeneration of vision, and be a potential new treatment option for Stargardt disease.
斯塔加特病或青少年黄斑变性是一种罕见的遗传性疾病，由ABCA4基因突变引起，导致黄斑变性和中心视力丧失。ABCA4基因突变会导致脂褐素积聚，脂褐素是受损血细胞分解和吸收后残留的物质。这种脂褐素的积聚导致光感受器细胞死亡，随后黄斑变性。由于斯塔加特病的性质，目前没有治愈方法或治疗选择。然而，近年来，利用干细胞疗法作为斯塔加特病潜在治愈方法和治疗手段的研究和探索有所增加。眼科领域越来越多的研究发现，使用干细胞有可能替代光感受器细胞的损失，减缓视力退化的进程，并成为斯塔加特病潜在的新治疗选择。
