Affiliations: Department of Radiology, University of Massachusetts, Worcester, MA, USA | Department of Neurosurgery, University of Massachusetts, Worcester, MA, USA | Departments of Microbiology & Physiological Systems, Immunology & Virology, Molecular Genetics & Microbiology, Gene Therapy Center, and RNA Therapeutics Institute, University of Massachusetts, Worcester, MA, USA | Department of Medicine, Neuro-therapeutics Institute and RNA Therapeutics Institute, University of Massachusetts, Worcester, MA, USA | Department of Animal Medicine, University of Massachusetts, Worcester, MA, USA | Department of Neurology, University of Massachusetts, Worcester, MA, USA | Departments of Pediatrics and Gene Therapy Center, University of Massachusetts, Worcester, MA, USA
Note: [] Correspondence to: Neil Aronin, University of Massachusetts Medical School, 364 Plantation Street, LRB-225, Worcester, MA 01605, USA. Tel. +1 508 856 3239; E-mail: Neil.Aronin@umassmed.edu
Abstract: Our goal is delivery of a long-term treatment for Huntington's disease. We administer intracerebrally in sheep adeno-associated virus (AAV) to establish optimal safety, spread and neuronal uptake of AAV based therapeutics. Sheep have large gyrencephalic brains and offer the opportunity to study a transgenic Huntington's disease model. However, lack of a relevant brain stereotactic atlas and the difficulty of skull fixation make conventional stereotaxy unreliable. We describe a multi-modal image-guidance technique to achieve accurate placement of therapeutics into the sheep striatum.
