Lysosomal Diseases: Stepping Stones to Gene Therapy for Alzheimer’s?
While gene-based therapy for late-onset AD may seem distant, rare neurological disorders could point the way.
While gene-based therapy for late-onset AD may seem distant, rare neurological disorders could point the way.
As gene therapy is making a comeback, scientists are exploring if it might prevent or reverse Alzheimer’s. Some of those treatments are permanent, heightening safety concerns.
Encouraged by success in treating infant spinomuscular atrophy, researchers are redoubling their efforts to target genetic causes of age-related neurodegeneration.
Specific patterns of expression defined distinct subtypes of neurons, astrocytes, oligodendrocytes, and microglia in this early affected brain region.
Brain biopsy tissue reveals that their transcriptomes shift by location, age, and disease.
In a mouse tauopathy model, knocking out the NLRP3 inflammasome prevented toxic tau from forming.
Based on preclinical data, researchers gave this antibiotic a shot in a two-year clinical trial. It did nothing to slow cognitive decline.
Cataloguing enhancer-promoter interactions in the four major cell types of the brain, researchers found that Alzheimer’s risk variants predominantly appeared in microglial enhancers.
Alzforum encourages users to visit the Virtual Exhibit Hall, where companies showcase their newest initiatives, products, and services. We welcome F. Hoffmann-La Roche, joining our other exhibitors — Biogen, BioLegend, Dash Genomics, Inc., Abcam, BrainXell, and the Jackson Laboratory.
The success of using gene therapy to treat spinal muscular atrophy in babies has researchers eyeing neurodegeneration as their next target. From suppressing Aβ production to improving immunotherapy to regenerating lost neurons, the field is flush with new approaches. Could a single injection of billions of viral capsids slow or prevent Alzheimer’s, Parkinson’s, and other dreaded diseases?
Different cell types respond to Alzheimer’s disease in distinct ways, but it has been difficult to measure this in the human brain. Now, a single-cell RNA analysis from postmortem entorhinal cortices of Alzheimer’s disease and control brain charts cell-type specific gene expression. The data provide new clues to cell types affected by genetic risk factors, and how they might contribute to disease.
New in last 7 days
Latest Comments