title: Human striatum remodelling after neurotransplantation in Huntington's disease.
abstract: BACKGROUND
  Restoration of functions in Huntington's disease (HD) by neurotransplantation stems from the formation of a striatum-like structure capable of establishing host connections as a result of grafted striatal neuroblast maturation. For the first time, we demonstrated some developmental steps accomplished by progenitor cells in the brain of an HD patient and analysed the molecular asset of the human primordium.
  
  
  METHODS
  Surgery involved bilateral (two sessions) stereotactic, caudate-putaminal transplantation of whole ganglionic eminence fragments from single legally aborted fetuses. MRI showed that the tissue deposits of the left hemisphere grew and joined to constitute a single tissue mass that remodelled basal ganglia anatomy and remained stable in size over time. No evidence of graft growth was observed contralaterally. PET demonstrated increased striatal and stable cortical metabolism. Unified Huntington's Disease Rating Scale assessments demonstrated improvement of motor performances, which faded over the 36-month follow-up. Cognitive performance tended to decrease at a lower rate than before transplantation.
  
  
  CONCLUSIONS
  The striatal primordium grew into the host brain and this process was associated with metabolic change and some clinical benefit. The study suggests the plasticity and reparative potential of un-manipulated primordium in an era where promising cell-based therapies are still in their infancy.
authors.affiliation: Careggi University Hospital, Florence, Italy.
authors.forename: Pasquale
authors.initials: P
authors.lastname: Gallina
authors.forename: Marco
authors.initials: M
authors.lastname: Paganini
authors.forename: Annibale
authors.initials: A
authors.lastname: Biggeri
authors.forename: Mirca
authors.initials: M
authors.lastname: Marini
authors.forename: Annamaria
authors.initials: A
authors.lastname: Romoli
authors.forename: Erica
authors.initials: E
authors.lastname: Sarchielli
authors.forename: Valentina
authors.initials: V
authors.lastname: Berti
authors.forename: Elena
authors.initials: E
authors.lastname: Ghelli
authors.forename: Carmela
authors.initials: C
authors.lastname: Guido
authors.forename: Letizia
authors.initials: L
authors.lastname: Lombardini
authors.forename: Benedetta
authors.initials: B
authors.lastname: Mazzanti
authors.forename: Paolo
authors.initials: P
authors.lastname: Simonelli
authors.forename: Alessandro
authors.initials: A
authors.lastname: Peri
authors.forename: Mario
authors.initials: M
authors.lastname: Maggi
authors.forename: Berardino
authors.initials: B
authors.lastname: Porfirio
authors.forename: Nicola
authors.initials: N
authors.lastname: Di Lorenzo
authors.forename: Gabriella B
authors.initials: GB
authors.lastname: Vannelli
mesh_terms: D000328:Adult; D016380:Brain Tissue Transplantation / Q000379:methods; D002491:Central Nervous System Agents / Q000627:therapeutic use; D003072:Cognition Disorders / Q000209:etiology; D003131:Combined Modality Therapy; D003342:Corpus Striatum / Q000000981:diagnostic imaging / Q000473:pathology*; D016332:Fetal Tissue Transplantation / Q000379:methods; D005500:Follow-Up Studies; D020869:Gene Expression Profiling; D006085:Graft Survival; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy / Q000473:pathology / Q000523:psychology / Q000601:surgery*; D007558:Italy; D008279:Magnetic Resonance Imaging; D008297:Male; D059906:Neuroimaging; D009473:Neuronal Plasticity; D049268:Positron-Emission Tomography; D012371:Robotics; D012720:Severity of Illness Index; D013238:Stereotaxic Techniques; D013687:Telencephalon / Q000196:embryology / Q000378:metabolism / Q000637:transplantation*
chemical_list: D002491:Central Nervous System Agents