A signature of Alzheimer’s disease is degeneration of brain cells due to amyloid plaques, large conglomerates of misfolded proteins. The Harvard Gazzette reports on recent advances in treating the disease from Dr. Dennis Selkoe’s laboratory.
First the researchers induced an animal model of Alzheimer’s in mice. Then they extracted cells from the mice and genetically modified them to include a gene for neprilysin, an enzyme that degrades the plaques. Then they re-introduced the modified cells into the brain.
This form of gene therapy is more promising than introducing genes with a virus because it carries little risk of causing tumors. The efficacy of this technique has yet to be seen in humans. Furthermore, it remains unclear whether degrading the plaques actually prevents the neurocognitive decline associated with the disease.