Department of Experimental Medicine and Biochemical Sciences, University of Perugia, 06126 Perugia, Italy.
J Control Release. 2013 Jan 10;165(1):75-81. doi: 10.1016/j.jconrel.2012.08.028. Epub 2012 Sep 3.
Recombinant human IGF-1 currently represents the only available treatment option for the Laron Syndrome, a rare human disorder caused by defects in the gene encoding growth hormone receptor, resulting in irreversibly retarded growth. Unfortunately, this treatment therapy, poorly impacts longitudinal growth (13% in females and 19% in males), while burdening the patients with severe side effects, including hypoglycemia, in association with the unfair chore of taking multiple daily injections that cause local intense pain. In this study, we have demonstrated that a single intraperitoneal graft of microencapsulated pig Sertoli cells, producing pig insulin-like growth factor-1, successfully promoted significant proportional growth in the Laron mouse, a unique animal model of the human Laron Syndrome. These findings indicate a novel, simply, safe and successful method for the cell therapy-based cure of the Laron Syndrome, potentially applicable to humans.
目前，重组人生长激素 IGF-1 是 Laron 综合征的唯一治疗选择。Laron 综合征是一种罕见的人类疾病，由生长激素受体基因缺陷引起，导致生长不可逆地迟缓。不幸的是，这种治疗方法对纵向生长的影响很小（女性为 13%，男性为 19%），同时还使患者承受严重的副作用，包括低血糖，以及每天多次注射带来的局部剧烈疼痛等不公平的负担。在这项研究中，我们证明了单次腹腔内植入微囊化猪支持细胞可以成功地促进 Laron 小鼠（人类 Laron 综合征的独特动物模型）的显著比例生长。这些发现表明，针对 Laron 综合征的细胞治疗的治愈方法具有新颖性、简单性、安全性和成功性，可能适用于人类。
