Retinitis pigmentosa (RP) is a hereditary retinal degeneration disease with no effective therapeutic approaches. Inflammatory and immune disorders are thought to play an important role in the pathogenesis of RP. Human umbilical cord mesenchymal stem cells (UCMSCs), with multiple biological functions such as anti-inflammation and immunoregulation, have been applied in different systemic diseases. We conducted a phase I/II clinical trial aiming to evaluate the safety and efficacy of intravenous administration of UCMSCs in advanced RP patients. All 32 subjects were intravenously infused with one dose of 10⁸ UCMSCs and were followed up for 12 months. No serious local or systemic adverse effects occurred in the whole follow-up. Most patients improved their best corrected visual acuity (BCVA) in the first 3 months. The proportions of patients with improved or maintained BCVA were 96.9%, 95.3%, 93.8%, 95.4%, 90.6%, and 90.6% at the 1st, 2nd, 3rd, 6th, 9th, and 12th month follow-up, respectively. Most of the patients (81.3%) maintained or improved their visual acuities for 12 months. The average NEI VFQ-25 questionnaire scores were significantly improved at the third month (P < 0.05). The average visual field sensitivity and flash visual evoked potential showed no significant difference (P = 0.185, P = 0.711). Our results indicated that the intravenous infusion of UCMSCs was safe for advanced RP patients. Most of the patients improved or maintained their visual functions in a long term. The life qualities were improved significantly in the first 3 months, suggesting that the intravenous infusion of UCMSCs may be a promising therapeutic approach for advanced RP patients.

中文翻译：

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脐带间充质干细胞的静脉输注维持并部分改善晚期色素性视网膜炎患者的视觉功能。

视网膜色素变性 (RP) 是一种遗传性视网膜变性疾病，没有有效的治疗方法。炎症和免疫紊乱被认为在 RP 的发病机制中起重要作用。人脐带间充质干细胞（UCMSCs）具有抗炎、免疫调节等多种生物学功能，已应用于不同的全身性疾病。我们进行了一项 I/II 期临床试验，旨在评估静脉注射 UCMSCs 在晚期 RP 患者中的安全性和有效性。所有 32 名受试者均静脉注射一剂 10 ⁸UCMSCs 并随访了 12 个月。整个随访过程中未发生严重的局部或全身不良反应。大多数患者在前 3 个月内改善了他们的最佳矫正视力 (BCVA)。在第 1、2、3、6、9 和 12 个月的随访中，BCVA 改善或维持的患者比例分别为 96.9%、95.3%、93.8%、95.4%、90.6% 和 90.6%。大多数患者 (81.3%) 的视力保持或改善了 12 个月。NEI VFQ-25问卷平均得分在第三个月显着提高（P  < 0.05）。平均视野敏感度和闪光视觉诱发电位无显着差异（P  = 0.185，P = 0.711）。我们的结果表明，静脉输注 UCMSCs 对晚期 RP 患者是安全的。大多数患者长期改善或保持其视觉功能。前 3 个月生活质量显着改善，表明静脉输注 UCMSCs 可能是晚期 RP 患者的一种有前景的治疗方法。