Gene Therapy Clear Amyloid Plaques in Animal Trials

Filed in archive Drugs, Vaccines and Therapeutics , Gene Therapy on August 31, 2007

untreated brain
Photo: Plaques comprised of amyloid-beta are the hallmark pathology of Alzheimer's disease. In this study, the scientists used an amyloid-degrading enzyme to clear these amyloid cobwebs from the brain - as illustrated in these treated (right) versus untreated (left) brain images.

In a study published in PLOS, Harvard scientists report that the delivery of skin cells genetically engineered to produce neprilysin into the brain of mice has successfully cleared amyloid plaques associated with Alzheimer's Disease.

The Harvard team used skin cells from the animal's own body to introduce a gene for an amyloid-busting enzyme known as neprilysin. The skin cells, also known as fibroblasts, "do not form tumors or move from the implantation site," Hemming notes. "They cause no detectable adverse side effects and can easily be taken from a patient's skin." In addition, other genes can be added to the fibroblast-neprilysin combo, which will eliminate the implants if something starts to go wrong.

As to whether the technique will work in humans, the researchers say that the technique needs to be further refined considering the larger size of the human brain.

Source: Harvard News Office