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Journal of Neurosurgical Sciences 2011 December;55(4):371-81

language: English

Progress in restorative neurosurgery: human fetal striatal transplantation in Huntington’s disease. Reviews

Gallina P. 1, Paganini M. 2, Lombardini L. 3, Giordano G. 4, Mascalchi M. 4, Romoli A. M. 2, Ghelli E. 2, Porfirio B. 5, Vannelli G. B. 6, Di Lorenzo N. 1

1 Department of Neurosurgery, University of Florence, Careggi University Hospital, Florence, Italy;
2 Department of Neurology, University of Florence, Careggi University Hospital, Florence, Italy;
3 Department of Hematology, Centro Nazionale Trapianti, University of Florence, Careggi University Hospital, Florence, Italy;
4 Department of Neuroimaging, University of Florence, Careggi University Hospital, Florence, Italy;
5 Department of Medical Genetics and Immunogenetics, University of Florence, Careggi University Hospital, Florence, Italy;
6 Department of Anatomy, University of Florence, Careggi University Hospital, Florence, Italy


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The purpose of this paper was to offer a review of the rationale, methods, biological and clinical results of human fetal striatal transplantation (HFST) in the treatment of Huntington’s disease (HD). HD is a heritable neurodegenerative disease in which degeneration of neurons in the striatum leads to motor, psychiatric and cognitive deficits. The disease is progressive and inexorably lethal. At present there are no curative treatments for HD. A restorative therapy based on the intrastriatal transplantation of striatal neuroblasts taken from human fetus is currently being explored as potential treatment in selected HD patients. Pilot clinical trials of HFST have been started in few neurosurgery restorative centres. Results demonstrated that HFST is feasible and safe without relevant adverse effects; grafted neuroblasts survive, grow without evidence of neoplasia or teratoma, build new tissue with striatal-like imaging features, and move into the host brain towards short and long-distance cortical and sub-cortical targets. HFST delays disease progression and provides a period of improvement and stability. Even though larger-scale studies are still necessary to establish the true value of such a treatment, at this time, HFST represents a promising experimental therapy for patients with HD and one of the most interesting clinical application of restorative neurosurgery.

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pgallina@unifi.it