Adeito-associated virus vectors for gene therapy oi neurodegenerative disorders

Matthew I. During, Paola Leone

Research output: Contribution to journalArticlepeer-review

23 Scopus citations


Adeno-associated virus (MV) shows significant potential as a gene delivery system. Although it is ubiquitous in its distribution, with approximately 85% of the adult population in the United States seropositive for the virus (1), it has never been associated with clinical disease. Not only is MV non-pathogenic, but it can infect with high efficiency both dividing and terminally-differentiated cells, moreover the wild-type virus integrates into a specific chromosomal site 12-5]. It also has a broad host range and the virus is extremely resistant to environmental extremes. These characteristics make it particularly attractive as a gene delivery vehicle. As the enthusiasm driving the proliferation of clinical gene transfer protocols has dampened recently due to the lack of clinical success often reflecting immunogenicity and inefficiency of the gene transfer methods used in these trials, MV with minimal (if any) toxicity and high efficiency in a wide range of cells and tissues, may become the vector-of-cho/ce for many applications. There have been a number of comprehensive recent reviews of AAV biology [6-11] and this article specifically discusses recent advances in the use of AAV vectors with particular emphasis on AAV vectormediated in vivo gene transfer in the mammalian central nervous system.

Original languageEnglish (US)
Pages (from-to)292-300
Number of pages9
JournalClinical Neuroscience
Issue number5
StatePublished - 1995
Externally publishedYes

All Science Journal Classification (ASJC) codes

  • Neuroscience(all)
  • Neuropsychology and Physiological Psychology


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