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Anagrafe della Ricerca dell'Università degli Studi di Perugia.
EnglishRecombinant 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 irreversible 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 vexatious, 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.
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| Titolo: | Reversal of experimental Laron syndrome by xenotransplantation of microencapsulated porcine Sertoli cells |
| Autori: | |
| Data di pubblicazione: | 2013 |
| Rivista: | |
| Abstract: | 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 irreversible 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 vexatious, 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. |
| Handle: | http://hdl.handle.net/11391/964991 |
| Appare nelle tipologie: | 1.1 Articolo in rivista |
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