Neurological Diseases: Gene Therapy


Gene therapy is the use of nucleic acids as drugs. This type of genetic pharmacology can be used to treat either inherited (e.g. Huntington disease) or mainly sporadic (e.g. Parkinson disease) neurological diseases.

Keywords: viral vectors; neurodegeneration; brain tumors; clinical trials in neurology; growth factors


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Further Reading

Chiocca EA and Breakefield XO (eds.) (1998) Gene Therapy for Neurological Disorders and Brain Tumors. Totowa, NJ: Humana Press.

Friedmann T (ed.) (1999) The Development of Human Gene Therapy. Cold Spring Harbor, NY: Cold Spring Harbor Laboratory Press.

Lowenstein PR (2002) Immunology of viral‐vector mediated gene transfer into the brain: an evolutionary and developmental perspective. Trends in Immunology 23: 23–30.

Lowenstein PR (ed.) (2003) Special Issue: Immune responses to viral vextors. Gene Therapy (in press).

Lowenstein PR and Castro MG (2002) Progress and challenges in viral vector‐mediated gene transfer to the brain. Current Opinion in Molecular Therapy 4: 359–371.

Lysaght MJ and Aebischer P (1999) Encapsulated cells as therapy. Scientific American 280: 76–82.

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Lowenstein, PR, and Castro, MG(Jan 2006) Neurological Diseases: Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1038/npg.els.0005757]