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.
NEWS
Gene Therapies Enter Trials for Many Brain Pathologies—What about AD?
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.
Time to Try Again: Gene-Based Therapy for Neurodegeneration
Encouraged by success in treating infant spinomuscular atrophy, researchers are redoubling their efforts to target genetic causes of age-related neurodegeneration.
Single-Cell Expression Atlas Charts Changes in Alzheimer’s Entorhinal Cortex
Specific patterns of expression defined distinct subtypes of neurons, astrocytes, oligodendrocytes, and microglia in this early affected brain region.
The Human Brain Hosts a Menagerie of Microglia
Brain biopsy tissue reveals that their transcriptomes shift by location, age, and disease.
Microglia Inflammasome Stokes Tau Phosphorylation, Tangles
In a mouse tauopathy model, knocking out the NLRP3 inflammasome prevented toxic tau from forming.
Minocycline Does Not Work in Mild Alzheimer’s Disease
Based on preclinical data, researchers gave this antibiotic a shot in a two-year clinical trial. It did nothing to slow cognitive decline.
Cell-Specific Enhancer Atlas Centers AD Risk in Microglia. Again.
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.
Virtual Exhibit Hall
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.
SPOTLIGHT
Is Gene-Based Therapy the Answer to Neurodegenerative Disease?
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?
Single-Cell Expression Atlas of Alzheimer’s Entorhinal Cortex
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.
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