[Ex vivo gene therapy in the treatment of Parkinson s disease].
OBJECTIVE
Taking into account the growing development and application of in vivo and ex vivo gene therapy in neurodegenerative disorders we review this kind of therapy applications in Parkinson s disease.


METHODS
Gene therapy carried out to this illness includes the liberation of genes encoding biosynthetic enzymes for dopamine synthesis: tyrosine hydroxylase, AADC and GTP cyclohydrolase and neurotrophic factors like GDNF which promotes the survival and maintenance of dopamin rgic neurons. Ex vivo gene therapy allows the control of the gene transfer before implantation, however one of the fundamental problems of this procedure is given by the immunologic rejection, so the use of autologous sources is recommended.


CONCLUSIONS
Ex vivo gene therapy is advantageous in relation to in vivo gene therapy because it allows the control of gene transfer before the implantation; looking for cellular sources of neural origin or pluripotent stem cells which can be differenciated toward a wanted cellular type in order to achieve the structural and functional integration of the cells implanted in the central nervous system are recommended; however it becomes necessary the development of vectors of new generation to avoid biosafety problems involved in the gene therapy.
García-Miniet
R
R
Departmento de Biología Molecular, Centro Internacional de Restauracion Neurológica, CIREN. La Habana, Cuba. rocio@neubas.sld.cu
Alberti-Amador
E
E
Castellano-Ortega
M R
MR
D001142:Aromatic-L-Amino-Acid Decarboxylases / Q000235:genetics / Q000378:metabolism; D004298:Dopamine / Q000096:biosynthesis*; D006136:GTP Cyclohydrolase / Q000235:genetics / Q000378:metabolism; D015316:Genetic Therapy; D051100:Glial Cell Line-Derived Neurotrophic Factor; D006801:Humans; D009414:Nerve Growth Factors / Q000235:genetics / Q000378:metabolism; D010300:Parkinson Disease / Q000201:enzymology / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D014446:Tyrosine 3-Monooxygenase / Q000235:genetics / Q000378:metabolism
C495039:GDNF protein, human; D051100:Glial Cell Line-Derived Neurotrophic Factor; D009414:Nerve Growth Factors; D014446:Tyrosine 3-Monooxygenase; D006136:GTP Cyclohydrolase; D001142:Aromatic-L-Amino-Acid Decarboxylases; D004298:Dopamine