Antibodies reduced disease features in mice with C9orf72-related ALS and FTD
Original source
Antibody Therapy Targeting RAN Proteins Rescues C9 ALS/FTD Phenotypes in C9orf72 Mouse Model. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human, Mouse
Related topics
Researchers developed antibodies targeting RAN proteins produced by the C9orf72 genetic expansion. In cells and C9orf72 mouse models, the antibodies reduced these proteins and signs of toxicity, improved behaviour, reduced neuroinflammation and neurodegeneration, and extended survival.
Why this matters
The findings identify a possible treatment strategy for C9orf72-related ALS and frontotemporal dementia. The work was conducted in cells and genetically modified mice, so it does not yet show a benefit for people or change current treatment.
Limitations and context
This was preclinical research in cells and C9orf72 BAC transgenic mice, not a human clinical trial. The results still need to be tested for safety and effectiveness in people with C9orf72-related disease.