IngentaConnect Adeno-associated virus vectors for gene transfer to the brain

Authors: Okada T.¹; Nomoto T.¹; Shimazaki K.²; Lijun W.^{1, 3}; Lu Y.^{1, 3}; Matsushita T.¹; Mizukami H.¹; Urabe M.¹; Hanazono Y.¹; Kume A.¹; Muramatsu S-i.³; Nakano I.³; Ozawa K.¹

Abstract:

Gene therapy is a novel method under investigation for the treatment of neurological disorders. Considerable interest has focused on the possibility of using viral vectors to deliver genes to the central nervous system. Adeno-associated virus (AAV) is a potentially useful gene transfer vehicle for neurologic gene therapies. The advantages of AAV vector include the lack of any associated disease with a wild-type virus, the ability to transduce nondividing cells, the possible integration of the gene into the host genome, and the long-term expression of transgenes. The development of novel therapeutic strategies for neurological disorder by using AAV vector has an increasing impact on gene therapy research. This article describes methods that can be used to generate rodent and nonhuman primate models for testing treatment strategies linked to pathophysiological events in the ischemic brain and neurodegenerative disorders such as Parkinson’s disease.

Language: English

Document Type: Research article

DOI: 10.1016/S1046-2023(02)00228-1

Affiliations: 1: Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical School, 3311-1 Yakushiji, Minami–Kawachi, Kawachi, Tochigi 329-0498, Japan 2: Department of Physiology, Jichi Medical School, Minami–Kawachi, Tochigi 329-0498, Japan 3: Department of Neurology, Jichi Medical School, Minami–Kawachi, Tochigi 329-0498, Japan