简单说明
Parkinson's disease develops when the brain cells that produce dopamine gradually die, causing tremor, stiffness and slowness of movement. For the great majority of people there is no single faulty gene to correct: the disease emerges from a mix of many small genetic influences, age and environment. Gene editing is a tool for changing specific sequences, and where no specific sequence is responsible it has very little to work with.
深入了解
Most Parkinson's disease is idiopathic and polygenic. A minority of cases involve variants in LRRK2, GBA1, SNCA, PRKN or PINK1, and those subgroups are where sequence-targeted approaches are conceivable. Gene editing is mainly used in Parkinson's research to build isogenic cell models — introducing or correcting a variant in otherwise identical stem cells to establish what it actually does. Cell-replacement therapies using stem-cell-derived dopaminergic neurons are in trials, but that is cell therapy rather than editing.
Why this page says no rather than soon
It would be easy to write an optimistic page here, because Parkinson's is common and gene editing is powerful. That would be misleading. Editing needs a target sequence whose change causes the disease. For most Parkinson's there is no such sequence. The honest statement is that editing is a research tool in Parkinson's today, and that the therapeutic work worth watching is cell replacement, which is a different technology.
Sources
- National Institute of Neurological Disorders and Stroke
Parkinson's disease ↗