Improved motor function and neuropathology
In an HMSN-P mouse model, the research group reported preservation of motor axons, improved motor performance, and extended survival.
- Disease
- HMSN-P
- Target
- TFG P285L variant
- Approach
- AAV-delivered adenine base editing
- Stage
- Basic / preclinical research
Reduced protein aggregation and neuronal death
In neuromuscular organoids generated from patient-derived iPS cells, the research group reported reduced abnormal TFG protein aggregation and neuronal death.
RESEARCH STAGE
Current research stage
These findings represent proof of concept in animal and human cell-derived models. The approach is not yet a treatment available to patients.
Further work required
- Safety and off-target assessment
- Optimization of administration and delivery
- Long-term efficacy and durability
SOURCE
September 18, 2026 — Center for iPS Cell Research and Application (CiRA), Kyoto University
This page summarizes publicly available academic research. It does not indicate that a company-owned product is in clinical development or that safety and efficacy have been established in patients.