Huntington's Disease (HD)
Patient Condition
Early Huntington's disease, CAG repetition number 45, mild chore-like movement, normal cognitive function, MoCA score 26. UHDRS TMS 14 points, TFC 13 points. Selected into the CRISPR gene editing treatment phase I clinical trial.
Treatment Timeline
| Phase | Details | Date |
|---|---|---|
| Clinical trial screening and baseline assessment | ||
| AAV-CRISPR gene therapy infusion | ||
| Postoperative recovery + immune management | ||
| Follow-up assessment and rehabilitation |
Treatment Outcome
| before | UHDRS TMS 14, TFC 13, MoCA 26, mild choreo-like movements, normal cognitive function |
|---|---|
| after | UHDRS TMS dropped to 6 points (improved by 8 points), TFC remained at 13 points, MoCA increased to 28 points, choreo-like movements almost disappeared, and cerebrospinal fluid mHTT protein levels dropped by 40% |
| highlight | CRISPR gene editing treatment phase 1 trial was safe, mHTT protein levels were significantly reduced, and exercise symptoms were almost completely controlled |