CAMBRIDGE, Mass., May 22 (UPI) -- U.S. biotech firm Genzyme said Tuesday its gene therapy approach showed promise for treating a deadly metabolic disease in a mouse model.
Genzyme said its gene-therapy technique helped preserve brain function and extend lifespan in mice with the equivalent of Niemann-Pick disease, a metabolic illness that in some forms can be fatal in infancy. The study appears in the May 22 issue of The Proceedings of the National Academy of Sciences.
The Genzyme researchers wrote in the journal that the gene therapy technique could be a promising strategy for treating diseases that affect organs and the central nervous system.
In the study, the Genzyme researchers injected adeno-associated viral vectors encoding an enzyme deficient in Niemann-Pick disease patients into the brain and body of mice. The animals that received both injections fared better than those receiving only one injection or those that were untreated.
The mice receiving the combination injections maintained motor and cognitive functions at near normal levels for 54 weeks and lived longer than the other two groups of animals.
