Bhat Mohd Akbar, Goyal Shiwali
Department of Ophthalmology, Georgetown University Medical Center, Washington, DC, USA.
Ophthalmic Genetics and Visual Function Branch, National Eye Institute/National Institutes of Health, Rockville, MD, USA.
Stem Cell Rev Rep. 2025 Jul 17. doi: 10.1007/s12015-025-10927-3.
Retinal degenerative diseases (RDDs) are a major global cause of irreversible vision loss, primarily resulting from the progressive degeneration of photoreceptors (PRs), retinal pigment epithelium (RPE), and retinal ganglion cells (RGCs). The limited regenerative capacity of the neural retina, combined with a lack of definitive therapies, highlights the urgent need for clinically viable strategies to slow degeneration or replace lost cells. While effective clinical treatments remain unavailable, recent advances in gene and stem cell therapies offer promising avenues to restore retinal structure and function. Preclinical and clinical studies have demonstrated encouraging safety and efficacy outcomes, supporting their potential to treat both inherited and acquired forms of RDDs. Nonetheless, several challenges-including vector limitations, immune responses, and delivery constraints-continue to hinder widespread clinical adoption. This review summarizes current gene and stem cell-based therapeutic strategies, recent clinical progress, and the key challenges and future directions shaping the evolving landscape of regenerative treatment for RDDs.
视网膜退行性疾病（RDDs）是全球不可逆视力丧失的主要原因，主要是由光感受器（PRs）、视网膜色素上皮（RPE）和视网膜神经节细胞（RGCs）的进行性退化引起的。神经视网膜有限的再生能力，加上缺乏明确的治疗方法，凸显了迫切需要临床上可行的策略来减缓退化或替代丢失的细胞。虽然仍然没有有效的临床治疗方法，但基因和干细胞疗法的最新进展为恢复视网膜结构和功能提供了有希望的途径。临床前和临床研究已经证明了令人鼓舞的安全性和疗效结果，支持了它们治疗遗传性和获得性RDDs的潜力。尽管如此，包括载体限制、免疫反应和递送限制在内的几个挑战继续阻碍其在临床上的广泛应用。本综述总结了当前基于基因和干细胞的治疗策略、最近的临床进展以及塑造RDDs再生治疗不断发展格局的关键挑战和未来方向。
