Genetic tools are getting more efficient and cheaper to use. CRISPR and sequencing technologies are very easy to use and relatively economical too. A few studies and clinical trials have focused on treating diseases by utilizing such tools as vectors to edit genomes of patients. Most of them had great results but only in cell cultures or animal models. If though this method for gene therapy proves to be safe and effective it may change medicine in a big way.
Alzheimer’s comes from the accumulation of amyloid beta (Aβ) protein in the brain. Tau phosphorylation may also be a cause leading to NTFs (neurofibrillary tangles) that eventually like amyloid beta particles accumulate and cause brain cells to lose their function. The results of those abnormalities is cognitive decline. Symptoms and causes though vary. Alzheimer’s specifically has been associated with certain genes.
Now researchers try to make gene therapies targeting those genes that appear to be causing most of the issues associated with Alzheimer’s disease. This study focused on the APPswe (Swedish) mutation in the APP gene that results in abnormally high concentrations of amyloid-β. By using CRISPR this study induced deletions in the APP gene attempting to reduce its negative effects. This showed a 60% reduction in Aβ. This though was the result in vitro.
When the researchers tried to transform brain cells of live mice the efficiency of the technique appears small. Still this may have been caused due to the number of copies of the gene present in each cell. It certainly is a proof of concept but it is also clear that we need to adjust our methodology to make gene therapy more effective in vivo.