GABA能祖细胞移植治疗难治性癫痫的研究进展
Advances in GABAergic Progenitor Cells Transplantation for Drug-Resistant Epilepsy
DOI: 10.12677/acm.2025.15123598, PDF,    科研立项经费支持
作者: 王小锐:西安医学院研究生处,陕西 西安;张 欣, 江 文, 孙中轻*:空军军医大学第一附属医院神经内科,陕西 西安
关键词: 癫痫GABA能祖细胞GABA能中间神经元细胞移植Epilepsy GABAergic Progenitor Cells GABAergic Interneurons Cell Transplantation
摘要: 癫痫是常见的慢性疾病,全球约≥5000万患者,对公共卫生与社会功能造成影响显著。在其病理生理机制中,兴奋性与抑制性神经传递失衡尤为关键,其中以抑制性γ-氨基丁酸(GABA)能中间神经元的减少或功能受损最具代表性。近年来,基于干细胞的修复策略备受关注。将多能干细胞分化得到的GABA能祖细胞植入癫痫相关脑区,可恢复局部的兴奋–抑制平衡,降低癫痫发生频率和程度。近期,一项针对癫痫患者、采用GABA能祖细胞移植的开放标签多中心I/II期临床研究(NCT05135091)亦呈现出良好的安全性和初步疗效。
Abstract: Epilepsy is a prevalent chronic neurological disorder affecting more than 50 million individuals worldwide, posing a substantial burden on public health and societal functioning. A central pathophysiological mechanism underlying epilepsy is the disruption of excitatory-inhibitory neurotransmission, prominently characterized by the loss or dysfunction of inhibitory γ-aminobutyric acid (GABA)-ergic interneurons. In recent years, stem cell-based regenerative strategies have received increasing attention. Transplantation of GABAergic progenitors differentiated from pluripotent stem cells into epileptogenic brain regions has been shown to restore local excitatory-inhibitory balance and reduce both the frequency and severity of seizures. Notably, an open-label, multicenter phase I/II clinical trial (NCT05135091) investigating GABAergic progenitor transplantation in patients with epilepsy has also reported favorable safety and promising preliminary efficacy.
文章引用:王小锐, 张欣, 江文, 孙中轻. GABA能祖细胞移植治疗难治性癫痫的研究进展[J]. 临床医学进展, 2025, 15(12): 1823-1829. https://doi.org/10.12677/acm.2025.15123598

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