基因疗法可能有助于治疗帕金森氏综合症
http://www.100md.com
2000年10月27日
NEW YORK (Reuters Health) - Results of a study in monkeys suggest that scientists may one day use gene therapy to halt the progression of Parkinson's disease. The therapy would restore the brain's ability to produce the chemical dopamine, effectively stopping the brain-cell degeneration that marks the disease.
Parkinson's disease is a disorder of the motor system in which the functioning of dopamine-producing brain cells becomes impaired. Dopamine is key to muscle control and movement. Parkinson's patients are currently treated with drugs that help replenish their brain's dwindling dopamine supply.
But researchers believe gene therapy can be used to deliver cell-repairing proteins to the brain's dopamine centers. Now, an international research team may have discovered how to do it. In the October 27th issue of Science, the group reports that gene therapy not only preserved dopamine cells in the brains of monkeys, but also reversed Parkinson's-like symptoms.
``This is the most potent (Parkinson's) therapy I've seen,'' lead author Jeffrey H. Kordower of Rush Presbyterian-St. Luke's Medical Center in Chicago, Illinois told Reuters Health in an interview.
He told Reuters Health that his team hopes to get the therapy into human trials after further research in monkeys.
Kordower and his colleagues delivered their therapy directly into ``vulnerable'' brain areas in monkeys that had a Parkinson's-like disorder. They injected a virus engineered to carry the gene for GDNF, a growth factor that stimulates dopamine cells. Once in the brain, the virus encoded the gene into dopamine-producing cells.
For humans, according to Kordower, the goal is a Parkinson's gene therapy in which a single treatment lasts for about 5 years. He noted that researchers still need to perfect a sort of automatic ``shut off'' for the GDNF gene so that it does not trigger an overproduction of dopamine.
In an editorial accompanying the report, Lars Olson of the Karolinska Institute in Stockholm, Sweden, writes that this study provides an ``optimistic outlook'' for treating Parkinson's, as well as other neurodegenerative diseases., http://www.100md.com
Parkinson's disease is a disorder of the motor system in which the functioning of dopamine-producing brain cells becomes impaired. Dopamine is key to muscle control and movement. Parkinson's patients are currently treated with drugs that help replenish their brain's dwindling dopamine supply.
But researchers believe gene therapy can be used to deliver cell-repairing proteins to the brain's dopamine centers. Now, an international research team may have discovered how to do it. In the October 27th issue of Science, the group reports that gene therapy not only preserved dopamine cells in the brains of monkeys, but also reversed Parkinson's-like symptoms.
``This is the most potent (Parkinson's) therapy I've seen,'' lead author Jeffrey H. Kordower of Rush Presbyterian-St. Luke's Medical Center in Chicago, Illinois told Reuters Health in an interview.
He told Reuters Health that his team hopes to get the therapy into human trials after further research in monkeys.
Kordower and his colleagues delivered their therapy directly into ``vulnerable'' brain areas in monkeys that had a Parkinson's-like disorder. They injected a virus engineered to carry the gene for GDNF, a growth factor that stimulates dopamine cells. Once in the brain, the virus encoded the gene into dopamine-producing cells.
For humans, according to Kordower, the goal is a Parkinson's gene therapy in which a single treatment lasts for about 5 years. He noted that researchers still need to perfect a sort of automatic ``shut off'' for the GDNF gene so that it does not trigger an overproduction of dopamine.
In an editorial accompanying the report, Lars Olson of the Karolinska Institute in Stockholm, Sweden, writes that this study provides an ``optimistic outlook'' for treating Parkinson's, as well as other neurodegenerative diseases., http://www.100md.com
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