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First published online October 27, 2004

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Gene therapy for prions

Transmissible spongiform encephalopathies (TSEs), such as Creutzfeld-Jakob disease and BSE, are thought to be caused by an abnormal, infectious form (PrP^Sc) of the normal cellular prion protein (PrP^C). PrP^Sc accumulates in the brains of infected individuals and seems to convert the endogenous PrP^C into more PrP^Sc. There is currently no cure for these diseases, but one idea is to take advantage of naturally occurring forms of PrP^C that cannot be converted to PrP^Sc. These can have dominant negative effects and could thus be used for gene therapy. On p. 5591, Véronique Perrier and co-workers show this approach holds promise. They demonstrate that lentiviral gene transfer can be used to deliver the PrP mutants Q167R and Q218K into chronically PrP^Sc-infected cultured cells. Using vectors derived from feline-immunodeficiency virus (FIV), they obtain an 80% transduction efficiency and show that the PrP mutants abolish replication of PrP^Sc in the cells. The authors go on to show that the FIV-derived vectors can efficiently deliver a ß-galactosidase marker to neurons and lymphoreticular cells in vivo. Since both are affected in TSEs, their results represent an important advance towards gene therapy for these diseases.

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Related articles in JCS:

Inhibition of PrP^Sc formation by lentiviral gene transfer of PrP containing dominant negative mutations

Carole Crozet, Yea-Lih Lin, Clément Mettling, Chantal Mourton-Gilles, Pierre Corbeau, Sylvain Lehmann, and Véronique Perrier
JCS 2004 117: 5591-5597. [Abstract] [Full Text]  

This Article Full Text (PDF) Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Related articles in JCS Similar articles in this journal Alert me to new issues of the journal Download to citation manager Citing Articles Citing Articles via Google Scholar Google Scholar Search for Related Content Social Bookmarking                         What's this?