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27 October 2000. Following on the heels of a report on a two-gene therapy approach to rescue neurons
in a mouse Parkinson's model, a new study published today in Science
finds that GDNF genes delivered by a lentiviral vector reversed functional deficits
and prevented neurodegeneration in a primate model. The lenti-GDNF was injected
directly into the striatum and substantia nigra, where it induced a high level
of GDNF expression. What's more, the introduced genes continued to express GDNF
for as long as eight months. Before introducing the gene therapy into humans,
there are several technical hurdles to surmount, notes Lars Olsen in an accompanying
Perspective. Methods to control the dose and expression level of the GDNF will
be important, Olsen notes, because excessive dopamine can result in ill effects,
including psychosis. It will also be important, he says, to detect Parkinson's
disease early enough while enough neurons remain to be rescued.-Hakon Heimer.
Reference:Kordower J, Emborg ME, Bloch J, Ma SY, Chu Y, Leventhal L, McBride J, Chen EY, Palfi S, Roitberg BZ, Brown WD, Holden JE, Pzyniski R, Taylor MD,
Carvey P, Ling ZD, Trono D, Hantraye P, Degion N, Aebischer P. Neurodegeneration
prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's
disease. Science 2000 Oct 27;290:767-772. Abstract
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Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease.
