title: Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference point.
abstract: OBJECTIVE
  To develop CERE-120 (AAV-NTN) as a novel therapy for Parkinson's disease (PD) that might restore function of degenerating dopamine neurons and prevent further degeneration.
  
  
  METHODS
  A nonclinical program demonstrated that NTN expression can be predictably controlled following CERE-120 administration, provides clear evidence of efficacy in numerous animal models and is safe at dose multiples that far exceed those required for efficacy. Preliminary, open label evidence in PD subjects offers corroborative support for these observations.
  
  
  CONCLUSIONS
  CERE-120 may represent an important, novel therapy for PD, though the clinical data require confirmation with additional clinical tests, including an ongoing multi-center, double-blinded controlled trial.
authors.affiliation: Ceregene, Inc., San Diego, CA 92121, USA.
authors.forename: Raymond T
authors.initials: RT
authors.lastname: Bartus
authors.forename: Christopher D
authors.initials: CD
authors.lastname: Herzog
authors.forename: Kathie
authors.initials: K
authors.lastname: Bishop
authors.forename: Jeffrey M
authors.initials: JM
authors.lastname: Ostrove
authors.forename: Mark
authors.initials: M
authors.lastname: Tuszynski
authors.forename: Jeffrey H
authors.initials: JH
authors.lastname: Kordower
authors.forename: Mehdi
authors.initials: M
authors.lastname: Gasmi
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D009479:Neuropeptides; D051101:Neurturin / Q000235:genetics / Q000502:physiology*; D010300:Parkinson Disease / Q000628:therapy*
chemical_list: C079279:CERE; D009479:Neuropeptides; D051101:Neurturin