title: Reversal of experimental Laron Syndrome by xenotransplantation of microencapsulated porcine Sertoli cells.
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 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.
authors.affiliation: Department of Experimental Medicine and Biochemical Sciences, University of Perugia, 06126 Perugia, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Luca
authors.forename: Mario
authors.initials: M
authors.lastname: Calvitti
authors.forename: Francesca
authors.initials: F
authors.lastname: Mancuso
authors.forename: Giulia
authors.initials: G
authors.lastname: Falabella
authors.forename: Iva
authors.initials: I
authors.lastname: Arato
authors.forename: Catia
authors.initials: C
authors.lastname: Bellucci
authors.forename: Edward O
authors.initials: EO
authors.lastname: List
authors.forename: Enrico
authors.initials: E
authors.lastname: Bellezza
authors.forename: Giovanni
authors.initials: G
authors.lastname: Angeli
authors.forename: Cinzia
authors.initials: C
authors.lastname: Lilli
authors.forename: Maria
authors.initials: M
authors.lastname: Bodo
authors.forename: Ennio
authors.initials: E
authors.lastname: Becchetti
authors.forename: John J
authors.initials: JJ
authors.lastname: Kopchick
authors.forename: Don F
authors.initials: DF
authors.lastname: Cameron
authors.forename: Tiziano
authors.initials: T
authors.lastname: Baroni
authors.forename: Riccardo
authors.initials: R
authors.lastname: Calafiore
mesh_terms: D000464:Alginates / Q000737:chemistry; D000818:Animals; D001835:Body Weight; D001846:Bone Development; D004195:Disease Models, Animal; D004339:Drug Compounding; D005260:Female; D020723:Glucuronic Acid / Q000737:chemistry; D006603:Hexuronic Acids / Q000737:chemistry; D007334:Insulin-Like Growth Factor I / Q000378:metabolism*; D046150:Laron Syndrome / Q000628:therapy*; D008297:Male; D051379:Mice; D008822:Mice, Transgenic; D011986:Receptors, Somatotropin / Q000235:genetics; D012708:Sertoli Cells / Q000637:transplantation*; D013552:Swine; D014183:Transplantation, Heterologous / Q000379:methods*
chemical_list: D000464:Alginates; D006603:Hexuronic Acids; D011986:Receptors, Somatotropin; D007334:Insulin-Like Growth Factor I; D020723:Glucuronic Acid