Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders.

Recombinant adeno-associated virus (rAAV) is derived from a small human parvovirus with an excellent safety profile. In addition, this viral vector efficiently transduces and supports long-term transgene expression in the nervous system. These properties make rAAV a reasonable candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five early stage clinical trials for various neurodegenerative disorders. Therefore, we will review the currently available preclinical data using rAAV in animal models of central nervous system (CNS) disorders. Moreover, potential caveats for rAAV-based gene therapy in the CNS are also presented.

• Publication year

  2006

• Venue

  Molecular Therapy

• Publication date

  2006-03-01

• Fields of study

  Medicine

• Identifiers
  DOI 10.1016/J.YMTHE.2005.11.009PMID 16412695
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

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